Saraswathi Vidya Bhawan’s College of Pharmacy, Dombivli (E)
The autoimmune disease known as type 1 diabetes mellitus (T1DM) is typified by the death of pancreatic ?-cells, which results in insulin insufficiency and a permanent reliance on exogenous insulin. One possible method for restoring endogenous insulin production and maybe curing type 1 diabetes is gene therapy. This tactic uses stem cell-based gene delivery, viral and non-viral vectors, and gene editing methods like CRISPR-Cas9 to either preserve already-existing ?-cells, replace lost ?-cells, or add insulin-producing cells from different sources. Recent developments concentrate on immune regulation to guarantee long-term safety and efficacy while halting more ?-cell death. Ongoing preclinical and clinical experiments are moving gene therapy closer to clinical implementation, despite obstacles like accurate gene targeting, immunological responses, and long-term integration. This review explores current progress, challenges, and future directions in gene therapy for T1DM, highlighting its potential as a transformative treatment.
Diabetes mellitus (DM) is apparently one of the primeval diseases commonly characterized by a rise in blood glucose levels that warrant constant monitoring and proper control (1). Pancreatic β-cells are responsible for production of hormone insulin which aid in absorption of glucose into cells in order to provide energy & is also engaged in other variety of functions. DM is triggered by insufficient insulin synthesis or sensitivity. Basically, there are 2 types of DM i.e Type 1 DM (T1DM) & Type 2 DM (T2DM) which are one of the most common types (1). T1DM is an autoimmune disorder which is represented by T-cell mediated self-destruction of insulin secreting islet β-cells in pancreas. T1DM has a complex root cause that can be caused due to genetic as well as environmental factors as compared to any other autoimmune diseases (1). Patients with T1DM has shown lower average life duration as compared to T2DM. It was found that patients with T1DM show elevated rate of CVS disorder & acute metabolic disorder. It is therefore crucial that all types of diabetes should be diagnosed & managed at initial phase to prevent or slow down its foreseeable challenges including other organs such as diabetic, nephropathy, retinopathy, neuropathy, CVS disorders & diabetic foot ulcer (2). Researchers have found that there are several techniques to manage complicated risks of diabetes, one such approach is Gene Therapy. Over the previous decades scientists have effectively discovered several genes that are obliged for evolution of T1DM (2). However, gene therapy is recently identified to have immense promise for diabetes management with ample of clinical studies which showed great safety & effectiveness for complex disorders (2). Gene therapy is a medical procedure used to treat or prevent disease by altering a person's genes. Gene therapy for Type 1 diabetes may entail modifying genes to restore insulin synthesis or controlling the immune system to safeguard beta cells that produce insulin (3). Cell-replacement therapy is the process of replacing damaged or non-functioning cells in the body by transplanting new cells, usually beta or stem cells. This usually entails replacing the insulin-producing beta cells that the immune system destroys in people with Type 1 diabetes (4). One potential option for treating type 1 diabetes that may satisfy all the requirements is gene therapy. The use of therapeutic genes as medications to treat an illness is known as gene therapy. To replace or restore lost biological capabilities, these therapeutic genes are transferred to target cells using a vector, such a virus (5). By converting non-β cells into surrogate β cells, avoiding the autoimmune destruction of β cells before the disease start, or simply replacing the function of lost β cells, therapeutic gene transfer can improve the clinical prognosis of diabetic persons with type 1 diabetes. Before delving deeply into the final option—replacing the function of β cells with insulin gene therapy—we will briefly outline the previous two gene therapy procedures and their drawbacks (5).
CAUSES
The exact cause of type 1 diabetes isn’t fully understood, but it generally starts when the body’s immune system mistakenly attacks and destroys the insulin-producing cells in the pancreas. The pancreas is an organ that plays a vital role in regulating blood sugar. When enough of these cells are damaged, the body can't produce enough, or any, insulin. This hormone helps glucose (a form of sugar) get into the cells where it’s used for energy.
Risk Factors for Type 1 Diabetes
While type 1 diabetes can affect anyone, some factors increase the likelihood of developing the condition:
- Family History: If a parent or sibling has type 1 diabetes, your chances of getting it are slightly higher.
-Genetics: Specific genes are linked to a higher risk.
Geography: People living farther from the equator are more likely to develop type 1 diabetes.
-Age: Type 1 diabetes can occur at any age, but it often appears in children between ages 4-7 a. (70)
COMPLICATIONS
If not carefully managed, type 1 diabetes can lead to serious complications that affect many parts of the body. Maintaining stable blood sugar levels can help reduce the risk of these complications:
Heart and Blood Vessel Disease: Type 1 diabetes increases the risk of heart-related problems, like coronary artery disease, heart attack, stroke, and high blood pressure.
Nerve Damage (Neuropathy): Consistently high blood sugar can damage tiny blood vessels that supply the nerves, particularly in the legs. This can cause symptoms like tingling, numbness, or pain in the feet and hands. Over time, this can lead to a loss of feeling, especially in the limbs.
Kidney Damage: Diabetes can damage the kidneys' filtering system, which can lead to kidney failure or the need for dialysis or a transplant.
Eye Damage: Diabetes can cause damage to the blood vessels in the retina, leading to diabetic retinopathy, which can result in blindness. It also increases the risk of other vision problems like cataracts and glaucoma.
Foot Damage: Poor circulation and nerve damage in the feet make it harder to notice injuries like cuts or blisters. If untreated, these can become infections, sometimes requiring amputation.
Skin and Mouth Conditions: People with diabetes are more prone to infections of the skin and mouth, such as bacterial or fungal infections. They may also experience gum disease and dry mouth.
Pregnancy Complications: If blood sugar is not well controlled during pregnancy, there can be a risk of miscarriage, stillbirth, birth defects, or complications like preeclampsia. The parent may also experience diabetic ketoacidosis and eye problems related to diabetes. (69)
Prevention of Autoimmune Beta Cell Destruction
To reduce the autoimmune onslaught and boost the survival of β cells, researchers have either overexpressed the antiapoptotic gene, bcl-2, in β cells specifically or overexpressed IL-4 via an adenoviral vector in nonobese diabetic mice. This approach depends on early diabetes identification, and by the time a person exhibits symptoms, more than 80% of their β cells have frequently already been damaged (56). It is practically impossible to determine whether a prediabetic person will eventually develop type 1 diabetes because the condition is complex. To produce replacement cells that are as close to natural β cells as possible, non-β cells are reprogrammed into surrogate β cells (57). The transformation of non-β cells into β cells has been shown to be dependent on the transcription factor pancreatic and duodenal homeobox gene 1 (PDX1), which controls β cell activity in adults and governs pancreatic development during embryogenesis (58). When PDX1 is expressed ectopically, non-β cells can be successfully transformed into β cells that can synthesize, process, and secrete insulin. Numerous investigations have revealed that reprogrammed surrogate β cells can help mice with streptozotocin-induced hyperglycaemia (59).
Immune System and Gene or Cell Replacement Therapy
Historically, insulin production in people with type 1 diabetes who have had their islets transplanted has been, at most, temporary. Auto implantation of islets taken from the patient's own pancreas following resection for chronic pancreatitis yields the best islet transplant outcomes. own pancreas following chronic pancreatitis resection (60) Given the long-term role of islets, it is possible that transplanted islets are harmed by existing immunosuppressive regimens. Naturally, the fact that the auto transplanted islets in these specific clinical situations are not only resistant to allorejection but also immune to the autoimmune attack that is the danger of type 1 diabetes makes it difficult to interpret these results (61). Clinical islet transplantation has been severely impeded by the intricacy of managing three distinct immunological attacks: acute rejection, chronic rejection, and disease recurrence. Since there isn't a single defective gene that has to be fixed, one could initially assume that gene therapy isn't appropriate for type 1 diabetes (62). However, it is undoubtedly an alluring idea to molecularly modify transplanted islets or cells to protect them from immune attack to prevent allorejection, disease recurrence, or both. It will be necessary to have well-regulated insulin secretion to treat type 1 diabetes with gene or cell replacement therapy without the requirement for any additional adjunct insulin therapy (63). Patients with type 1 diabetes will not benefit from constitutive secretion, regardless of the level of insulin gene transcription regulation that goes along with it. Lastly, it goes without saying that to treat type 1 diabetes, new and more abundant sources of cells that secrete insulin will be required (64). In this context, the most recent (albeit extremely early) advancements in stem cell research are encouraging, as islets or islet cells have been isolated from adult human pancreatic ductal stem cells as well as embryonal murine and human embryos (65).
1.Gene and Cell-Replacement Therapy in the Treatment of Type 1 Diabetes
1.1 Historical Perspective
The history of gene therapy and cell-replacement therapy for the treatment of Type 1 diabetes dates back many years. Initially, the only therapy was insulin. However, the discovery of genetic modification in the 1970s provided a new avenue for research. Then, in the 2000s, stem cell research brought new breakthroughs. Some of the most important milestones include the studies on beta cell transplantation and the use of gene therapy to assist the immune system in acceptin [5].
1.2 Minimal Standards and Expectations.
This includes ensuring that normal insulin production would be restored while stabilizing the level of glucose in the bloodstream without the host immune system having any reaction, like rejection to the new cells or requiring an antirejection treatment with stronger immunosuppression drugs, for the procedure to be beneficial for Type 1 diabetes sufferers to receive gene and cell-replacement therapy. At the same time, the therapeutic modality would also have to be safe as well as accessible to be available in a population scale. [6].
1.3 How Good is the β-Cell at Controlling Glycemia?
Beta cells are crucial in the regulation of blood sugar because they synthesize and release insulin whenever the blood sugar level increases. They are quite efficient at controlling the blood sugar level when functioning properly. However, when the cells are destroyed, as is the case in Type 1 diabetes, the body loses the mechanism to regulate the blood sugar levels. [7].
1.4 Integrated Stimulus-Secretion Coupling Circuitry in the β-Cell: A Tough Act to Follow
The system of beta cells regulating insulin release is complex and not easily replaced. Glucose is the main trigger for insulin secretion, but other substances like amino acids and fatty acids also help stimulate the beta cells. Hormones like GLP-1 and GIP also support insulin release, especially after eating. Finally, there are several processes in the beta cells that work together to produce the release of insulin. Some of them are the movement of calcium and potassium, which help enable the insulin to get released into the bloodstream. [8].
1.5 (Pro)Insulin Production: Keeping Pace with Secretion
The production of insulin must match the demand in the body, and beta cells do this in a very regulated manner. It is regulated at different levels to maintain the right balance. If blood sugar or other signals increase, beta cells respond by making more insulin mRNA, which leads to more insulin being produced. [9].
1.6 Kinetics of Insulin Production
Insulin production and release depend on various mechanisms that may vary. Beta cells rely primarily on regulated secretion, where insulin is released once blood sugar levels increase. At the same time, they continuously release a small, constant amount of insulin to maintain the basic insulin level in the body, known as constitutive secretion. [10].
1.7 Physiological and Clinical Considerations Related to Insulin Release in Humans: Must These Be Addressed?
? The release of insulin in the human body has some important considerations. First, there are two types of insulin secretion: basal and stimulated. Basal secretion occurs between meals to keep blood sugar stable, while stimulated secretion occurs after eating to handle the spike in blood sugar. [11].
? Physical activities and body weights also influence insulin requirements. Activities make the body more sensitive to insulin, therefore requires lower insulin. An overweight or an inactive person causes the body to be less responsive, thus require more insulin. As people get older, often the beta cells in the pancreas work less well, and in some cases, insulin sensitivity falls, making the blood sugar even harder to keep under control. [12].
? The mass of beta cells can vary depending on the body’s needs. For instance, it can increase during pregnancy or weight gain when the body requires more insulin. However, in cases such as Type 1 diabetes, autoimmune damage may reduce the number of beta cells. [ 13].
1.8 Are Animal Models Appropriate for Testing the Capability of Engineered β-Cells to Reverse Diabetes in Humans?
? When it comes to diabetes research, animals such as rodents have been incredibly useful. Many researchers use these animals to try out new engineered beta cells and gene therapies. It is an excellent starting point, but still a challenge: because these experiments work in animals, they might not work exactly the same in humans. The body has big differences, especially with regards to immune reactions and sugar-handling of the cells. [14].
? Although animal models give the researcher crucial insights—such as how to regrow beta cells or tweak the immune system—there is still a lot to consider before these treatments can be used on people. The gap between how animals and humans respond has to be figured out to make sure any therapy is actually effective and safe. [15].
1.9 “Clinical Shifting”—Switching One Clinical Outcome for Another
?Sometimes in diabetes therapy, focusing too much on a single health aim inadvertently generates another problem. Intensive insulin treatment to maintain strict blood sugar control usually results in weight gain. This is due to the reason that tighter control of glucose leaves less sugar within the urine. The body tends to retain more calories because there is less urine sugar loss. However, all this excess weight can make a body more resistant to insulin and complicate its management further. [16].
? There is also the correlation of insulin resistance with heart diseases. People diagnosed with Type 2 diabetes or insulin resistance will have a high level of proinsulin in their body; this is because proinsulin is linked with heart disease. While controlling the blood sugar levels is necessary, the long term use of insulin will lead to an increase in insulin resistance hence increasing the proinsulin which may cause heart problems. [17].
?There is also the issue of cell-replacement therapy. In order to prevent the body from rejecting these transplanted cells, patients have to be on immunosuppressive drugs. However, these drugs have severe side effects, such as the increased risk of cancer and other health issues. [18].
2. Insulin Gene Therapy for Type 1 Diabetes Mellitus
2.1 Introduction
2.1 Alright, so here’s the deal with Type 1 diabetes, or T1DM for short. It’s a condition where your immune system—a system that’s supposed to protect you—completely messes up. Instead of fighting off germs or viruses, it goes rogue and attacks the insulin-making beta cells in your pancreas. [19]
- This is a big problem because insulin is the hormone that keeps your blood sugar levels in check. When those beta cells are destroyed, you end up needing insulin injections for the rest of your life to keep things balanced. Now, traditional treatments like insulin shots or even getting a new pancreas for islet cells are out there, and yes, they do the job. To an extent [20]. - But they come with a bunch of not-so-great side effects and complications. That’s where the party gets exciting – scientists are working on this novel gene therapy involving insulin. Sounds a little crazy: instead of trying to revive all those beta cells, they can just manipulate the rest of the cells into making insulin. If this actually works, the management of Type 1 diabetes may be absolutely revolutionized for the better or at best serve as a practically painless avenue for living with this disease. [21].
2.2 Traditional Therapies for Treatment of Type 1 Diabetes Mellitus
Exogenous Insulin-Based Therapies: The most common treatment for Type 1 diabetes is the use of insulin injections or insulin pumps. These can keep blood sugar levels in check pretty well, but they do not treat the underlying problem-the destruction of the beta cells in the pancreas. People with T1DM continue to have to monitor their glucose levels and must adjust their levels of insulin delivery, which does get very repetitive. It’s also not something that replicates the natural role of a functioning pancreas regarding insulin regulation and therefore is less than ideal. [22].
- Transplant Therapies: The most extreme treatment would be to implant the entire pancreas or, at least the islet cells-the cells responsible for producing insulin. The proposal is to rejuvenate the demolished beta cells, and then to let the body regain its normal production of insulin. Sounds super, doesn’t it? However, this therapy has grave downsides. [23].
1. Donor Availability: There are not nearly enough donor organs or cells available to keep pace with the demand. That is a big problem. [24].
2. Transplant Rejection: The patient, after the transposition has to be placed under immunosuppressive drugs so that his body does not accept the new tissue. Again, these drugs also pose some problems and their consequences which go along with it. [25].
3. Long-Term Viability: Even if a transplant works initially, the transplanted pancreas or islets might not last forever. Over time, they can lose their function, and the patient might need another transplant or some other intervention to keep things going [26].
2.3 Gene Therapy for Treatment of Type 1 Diabetes Mellitus.
Gene therapy for Type 1 diabetes sounds like a great idea. What it does fundamentally is introduce into cells that aren’t beta cells a healthy copy of the insulin gene. With this, it will be producing insulin again from those cells. The hope in this is long-term insulin generation without the reliance on daily insulin shots. This is broken up into a couple of key points:
1. Gene Delivery Systems: Scientists use something called viral vectors to get the insulin gene into the appropriate cells. It’s like tiny delivery trucks – like adenoviruses or lentiviruses – which carry the gene into the target cells. [27].
2. Target Cells: Not all the cells are best suited for such work, hence it is left to select. Generally, Liver cells, termed hepatocytes have been preferred many a times since these cells sustain the gene to an extended duration, and even already participate in controlling glucose inside the body. [28].
2.4 Prevention of Autoimmune β Cell Destruction.
One of the biggest challenges with Type 1 diabetes is stopping the immune system from attacking the beta cells in the pancreas. For insulin gene therapy to really work, we need to prevent this destruction. Here are some strategies that might help:
? Immune Modulation: One way to tackle this is by using immunosuppressive drugs or therapies that modify or changes the immune system. These treatments aim to calm down the immune response and reduce its attack on the beta cells [29].
? Gene Editing: Another exciting approach is using gene editing tools like CRISPR/Cas9. This technology can either alter the immune system or even make the beta cells themselves less vulnerable to the immune systems attack. [30].
? Vaccination: Researchers are also looking into vaccines that could specifically target the autoimmune response against the beta cells. If successful, these vaccines could help prevent Type 1 diabetes from developing in the first place or stop it from getting worse once its already started [31].
2.5 Reprogramming Non-β Cells into β Cells
Reprogramming is a new concept involving the making of other types of cells into beta cells to produce insulin. Here is how it works:
- Cellular Reprogramming: It implies the conversion of other types of cells, including hepatocytes – liver cells- or pancreatic duct cells, such that they carry a beta-cell-like function. This is through the introduction of specific “instructions” or transcription factors that lead to these cells beginning to manufacture insulin. [32].
? Transdifferentiation: In this technique, it is possible to directly convert the cells into beta cells that will produce insulin without necessarily having to be involved with a stage of stem cells. This can be achieved through modulating gene expression to make the cells be programmed to become beta cells directly [33].
2.6 Insulin Gene Therapy
Insulin gene therapy is meant to introduce the insulin gene into hepatocytes, such as liver cells. This gene introduction will result in the possibility of these hepatocytes producing the protein again.
- Gene Delivery: Now we need to determine the most appropriate delivery vehicle in order to transfect the cells with the insulin gene. Viral vectors like adenovirus or lentiviruses have frequently been utilized; these might be thought of as small trucks to deliver the gene into target cells. [34].
- Regulation of Expression: Once the gene for insulin enters the cells, we have to ensure that the gene produces appropriate levels of insulin, just as a healthy pancreas would. This means tightly regulating how much insulin is produced and not too much or too little [35].
- Long-Term Stability: The idea is to ensure that the insulin gene will be functional for a long time. This implies ensuring that the altered cells keep on producing insulin with a minimal or no occurrence of immune reactions that could damage them. [36].
2.7 Future Perspectives
The future of insulin gene therapy holds a lot of promise, with several important areas that need to be addressed to improve its effectiveness:
? Improved Delivery Systems: One of the key challenges is creating better, safer ways to deliver gene editing tools and therapeutic genes to the right cells. More efficient delivery systems would increase the success rate of these therapies [37].
? Enhanced Targeting: It's crucial to develop more precise techniques to make sure the gene therapy reaches only the right cells. This would improve the efficiency and reduce potential side effects [38].
? Clinical Trials: As with any new treatment, we need thorough clinical trials to assess the safety, efficacy, and long-term outcomes of insulin gene therapy in humans. These trials will be essential for proving whether the therapy can be safely and effectively used in real-world settings [39].
? Combination Therapies: There’s also a growing interest in combining insulin gene therapy with other treatments, such as immune-modulating therapies or cellular reprogramming, to create a more comprehensive and effective approach to managing or even curing Type 1 diabetes [40].
3.Stem cell therapy for type 1 diabetes mellitus
3.1 Introduction.
This approach has developed into a new therapy that opens the possibility of changing the paradigm of Type 1 diabetes treatment by attacking the root cause of the condition, that is, beta cell loss. In Type 1 diabetes, the immune system mistakenly attacks and destroys the insulin-producing beta cells in the pancreas, leaving no production of insulin. [41].
Stem cells are a great hope in regenerating functional beta cells or even protecting the remaining ones from further immune attacks. Stem cell therapy, through restoration of natural insulin production and improvement of blood glucose regulation, may hold long-term prospects for cure or, at least, an acceptable management of Type 1 diabetes [42].
3.2 Many Roads Lead from Stem Cell to Beta Cells.
There are many pathways of producing beta cells from stem cells, and all have their pros and cons. Here is a glance at some of the promising sources:
- Human Embryonic Stem Cells (hESCs): Human embryonic stem cells are pluripotent; that is, they can be turned into any type of cell, including pancreatic beta cells. While the hESCs can be pushed into differentiating towards beta cells, which can now secrete insulin, thereby providing another potential therapeutic approach toward diabetes in a laboratory-based setting, there remains further ethical considerations associated with such use and clinical application is limited due to immune rejection because these are not the cells originating from the individual's own tissue [43].
- Induced Pluripotent Stem Cells (iPSCs): Induced pluripotent stem cells are the adult cells reprogrammed to resemble embryonic stem cells. Using iPSCs, one could obtain beta cells from a patient’s own tissue, for instance, from their skin or blood cells. That way, by using this system, many issues related to the use of hESCs may be circumvented, including those of ethics and immune rejection as the cells produced are genetically the same as that of the patients. [44]. This makes iPSCs a more promising option for treating Type 1 diabetes without the need for immunosuppressive drugs.
- Umbilical Cord Blood: Umbilical cord blood, collected after delivery, is made of hematopoietic stem cells that have been shown to turn into pancreatic cells. Although such cells are less heterogeneous than pluripotent stem cells, experiments have shown that cord blood cells could be differentiated into beta-like cells. This develops as another source of stem cells to treat diabetes. [45].
- Bone Marrow-Derived Mesenchymal Stromal Cells: MSCs are multipotent cells derived from the bone marrow that can differentiate into nearly any cell type, including beta cells. MSCs differ from pluripotent stem cells. The immune system would be likely to accept MSCs more readily, thereby eliminating the need for immunosuppressive therapy. MSCs also secrete factors that could be useful for the support of already existing beta cell regeneration and the modulation of immune responses, making them particularly beneficial for the treatment of Type 1 diabetes. [46].
- Organ-Specific Stem or Progenitor Cells: Pancreatic-specific stem cells, such as those originating from the ductal or acinar cells, are known to regenerate beta cells. The progenitor cells will certainly be a reliable source through which functionally active beta cells can be produced, though much of this work is still in infancy [47].
- Ductal Epithelial Cells: Other attractive sources are the pancreatic ductal epithelial cells. These pancreas duct-localized progenitors may be differentiable into functional beta cells. Beta cells release insulin. Thus, the inherent regeneration ability of the ductal cells forms the basis for this beta-cell regeneration technique. It is believed that such regeneration provides one of the critical means through which functional beta cells can be achieved for treatment purposes. [48].
- Acinar Cells: The acinar cells, which under normal conditions produce digestive enzymes, are currently being explored for their potential to be reprogrammed into insulin-producing beta cells via a process known as transdifferentiation. This may open another route for creating a sufficient number of beta cells for the treatment of diabetes. [49].
- Liver Cells: Researchers have also explored liver cells as a potential source of beta cells. In this regard, scientists hope that reprogramming of liver cells can exhibit characteristics similar to those of beta cells to make an accessible and viable source of insulin-producing cells. Since the tissue of liver is more easily available than the tissue of the pancreas, this may present a less invasive approach for therapies based on stem cells. [50].
3.3 Stem Cells and Autoimmunity
The biggest challenge we face in using stem cells to treat Type 1 diabetes is the body’s immune system, which tends to attack the beta cells. This doesn’t just affect the original beta cells; it can target any new ones that are introduced into the body as well. So, scientists have been working on several ways to try to prevent this from happening:
- Immune Modulation: The suppression of the immune system in order not to act against the transferred or regenerative beta cells, which also can be successful; however, as a consequence of this suppression, the body does not react too well against microbes and other invasive agents, factors that have to be handled quite carefully by the doctors. [51].
- Genetic modification: One more approach was genetic modification. The stem cells could be genetically modified before they are transplanted. Some genes can be modified so the stem cells will not be recognized by the immune system and hence will not be attacked. It is as if giving the new cells camouflage so they could do their work without being destroyed. [52].
- There is also the possibility of putting the beta cells in a protective casing. That would protect them from immune system attacks but allow them to still release insulin and exchange nutrients with the body. It’s a bit like giving the cells a safe space to function without worrying about the immune system stepping in. [53].
3.4 Stem Cell and Tumorigenesis.
Most dangers in therapies with the use of stem cells, especially in pluripotent cells, are the risk of tumours. In case these undifferentiated cells that originate from an embryo are put into action without becoming completely differentiated to become beta cells, they then remain undifferentiated and start to grow uncontrollably thus causing tumour[54].
- Researchers have faced this challenge by screening the stem cells in such a way that they become fully differentiated before their transplantation, so the incidence of tumors is highly reduced. It then guarantees that the treatment is both safe and efficient for patients. [55].
CONCLUSION:
In a nutshell, by having the possibility of replacing or modifying beta cells as part of a wider strategy in the agenda of stem cell therapy, the Type 1 diabetes condition may be solved. New advancements in the generation of insulin-producing cells from various stem cell sources, from human embryonic stem cells to induced pluripotent stem cells, and even more accessible sources such as umbilical cord blood and bone marrow, open new doors to future therapeutic possibilities. Of course, dangers inherent to any stem cell therapy, such as autoimmune destruction of beta cells and the risk of tumor formation, have to be addressed. Continued research and clinical trials are required to develop these therapies further, making them safe, effective, and accessible to patients
REFERENCE
Nikita Choudhary*, Jhanvi Yadav, Saujanya Rokade, Khushi Malve, Prerana Nyaynit, Maggie Jo Alex, Gene Therapy on Type 1 Diabetes Mellitus: An Overview of Contemporary Treatment Strategies for Type 1 Diabetes Mellitus, Int. J. Sci. R. Tech., 2025, 2 (4), 225-235. https://doi.org/10.5281/zenodo.15199137
10.5281/zenodo.15199137
