Gene therapy (जीन चिकित्सा) is a medical technology that aims to produce a therapeutic effect by manipulating gene expression or altering the biological properties of living cells. The first attempt at modifying human DNA was performed in 1980, by Martin Cline, but the first successful nuclear gene transfer in humans, approved by the National Institutes of Health, was conducted in May 1989. The first therapeutic use of gene transfer as well as the first direct insertion of human DNA into the nuclear genome was performed by French Anderson in a trial starting in September 1990. In 2003, Gendicine became the first gene therapy to receive regulatory approval.
Gene therapy Meaning
Gene therapy is a medical technique that involves modifying or manipulating an individual’s genetic material to treat or prevent disease. It aims to correct or replace defective or missing genes or introduce new genes into the body to help treat medical conditions.
Applications of Gene therapy
Gene therapy has many applications in medicine and biotechnology, aimed at treating, managing, or preventing various diseases. Here are some key areas where gene therapy is being applied or researched:
Genetic Disorders
Gene therapy addresses diseases caused by genetic mutations by correcting or replacing faulty genes. Examples include:
- Sickle Cell Anemia: Introducing healthy hemoglobin genes into bone marrow stem cells.
- Cystic Fibrosis: Delivering a functioning CFTR gene to lung cells.
- Hemophilia: Correcting or replacing genes responsible for clotting factors.
Cancer Treatment
Gene therapy can enhance the body’s ability to fight cancer by:
- CAR-T Cell Therapy: Engineering a patient’s T-cells to target and destroy cancer cells.
- Oncolytic Viruses: Using viruses modified to infect and kill cancer cells selectively.
- Gene Silencing: Turning off genes that promote tumor growth.
Neurological Disorders
Gene therapy is being explored to treat disorders of the nervous system, including:
- Parkinson’s Disease: Delivering genes that increase dopamine production.
- Huntington’s Disease: Silencing the mutated gene responsible for the disorder.
- Spinal Muscular Atrophy (SMA): Using therapies like Zolgensma to replace defective SMN1 genes.
Infectious Diseases
Gene therapy can enhance immune responses or directly target pathogens:
- HIV: Editing genes to make immune cells resistant to the virus (e.g. CCR5 gene modification).
- Hepatitis B: Targeting and silencing viral DNA within liver cells.
Cardiovascular Diseases
Gene therapy is used to address conditions like:
- Coronary Artery Disease: Promoting the growth of new blood vessels (angiogenesis).
- Heart Failure: Improving heart function by delivering therapeutic genes to cardiac tissue.
Ophthalmic Disorders
Some genetic forms of blindness are being treated with gene therapy:
- Leber Congenital Amaurosis (LCA): Introducing a functional RPE65 gene to restore vision.
- Retinitis Pigmentosa: Gene therapy approaches to slow or reverse degeneration.
Autoimmune and Inflammatory Diseases
Modifying immune cells or delivering anti-inflammatory genes can help manage conditions like:
- Rheumatoid Arthritis: Reducing inflammatory cytokines.
- Multiple Sclerosis: Enhancing regulatory immune functions.
Rare Diseases
Gene therapy has shown promise for treating many rare diseases, such as:
- Metachromatic Leukodystrophy (MLD): Replacing defective genes causing myelin damage.
- Wiskott-Aldrich Syndrome: Correcting genetic mutations in immune cells.
Regenerative Medicine
Gene therapy plays a role in tissue engineering and repairing damaged tissues:
- Wound Healing: Stimulating growth factors to enhance recovery.
- Bone Regeneration: Using genes to promote bone growth in orthopedic conditions.
Future and Emerging Applications
- Aging-Related Disorders: Targeting genes involved in aging processes.
- Metabolic Disorders: Managing diabetes or obesity through gene modifications.
- Biotechnology: Creating gene-based vaccines and enhancing agricultural productivity.
Gene therapy is a transformative field with the potential to revolutionize medicine, although challenges like delivery methods, long-term effects, and cost remain areas of focus for ongoing research.
List of diseases treated with Gene therapy
Gene therapy has been used to treat or is being researched for various diseases, including genetic, acquired, and complex conditions. Here’s a list of diseases treated or targeted by gene therapy:
Genetic Disorders
Mutations in specific genes cause these:
- Sickle Cell Anemia
- Beta-Thalassemia
- Cystic Fibrosis
- Duchenne Muscular Dystrophy (DMD)
- Spinal Muscular Atrophy (SMA)
- Severe Combined Immunodeficiency (SCID)
- “Bubble boy disease” (e.g. ADA-SCID)
- Hemophilia A and B
- Huntington’s Disease
- Leber Congenital Amaurosis (LCA)
- Wiskott-Aldrich Syndrome
- Metachromatic Leukodystrophy (MLD)
- Fabry Disease
- Tay-Sachs Disease
Cancer
Gene therapy is used to modify immune cells or target cancer cells:
- Leukemias (e.g. Acute Lymphoblastic Leukemia – ALL): CAR-T cell therapy.
- Lymphomas
- Multiple Myeloma
- Glioblastoma (brain cancer)
- Prostate Cancer
- Melanoma
- Non-Small Cell Lung Cancer (NSCLC)
Neurological Disorders
Gene therapy aims to address gene mutations or enhance neural function:
- Parkinson’s Disease
- Amyotrophic Lateral Sclerosis (ALS)
- Huntington’s Disease
- Rett Syndrome
- Canavan Disease
- X-linked adrenoleukodystrophy (ALD)
Ophthalmic Disorders
Gene therapy has shown success in treating inherited forms of blindness:
- Leber Congenital Amaurosis (LCA)
- Retinitis Pigmentosa
- Choroideremia
- Stargardt Disease
- X-Linked Retinoschisis
Infectious Diseases
Gene therapy helps in enhancing immunity or directly targeting pathogens:
- HIV/AIDS
- Hepatitis B Virus (HBV)
- Cytomegalovirus (CMV)
Cardiovascular Disorders
Gene therapy is used for angiogenesis or enhancing cardiac function:
- Coronary Artery Disease
- Heart Failure
- Peripheral Artery Disease (PAD)
- Familial Hypercholesterolemia
Autoimmune and Inflammatory Disorders
Gene therapy is under development for:
- Rheumatoid Arthritis
- Lupus
- Multiple Sclerosis
Rare Diseases
These often lack other effective treatments:
- Ornithine Transcarbamylase Deficiency (OTC)
- Glycogen Storage Disease (GSD)
- Pompe Disease
- Crigler-Najjar Syndrome
- Mucopolysaccharidosis (e.g. MPS I, II, III)
Skin Disorders
Gene therapy has been applied for:
- Epidermolysis Bullosa (EB): A genetic condition causing fragile skin.
- Wound Healing Disorders
Blood Disorders
Gene therapy addresses issues with blood production or function:
- Sickle Cell Disease
- Beta-Thalassemia
- Fanconi Anemia
Future Potential Areas
- Diabetes (e.g. regenerating insulin-producing cells)
- Aging-Related Diseases
- Obesity
- Neurodegenerative Disorders like Alzheimer’s Disease.
This list continually expands as gene therapy research advances, offering hope for previously untreatable diseases.
Gene therapy Classification
Gene therapy can be classified based on various criteria, including the target cells, delivery methods, and therapeutic approach. Here’s an overview of the main classifications:
1. Based on Target Cells
Somatic Gene Therapy
- Targets somatic (non-reproductive) cells.
- Changes are not inherited by offspring.
- Used to treat diseases in the individual, such as cancer, hemophilia, or sickle cell anemia.
Germline Gene Therapy
- Targets germline (sperm or egg) cells.
- Changes are heritable and passed to future generations.
- Raises ethical concerns and is not widely practiced in humans.
2. Based on Therapeutic Approach
Addition Therapy
- Introduces a functional gene to replace or supplement a defective or missing gene.
- Example: Adding a healthy CFTR gene for cystic fibrosis.
Replacement Therapy
- Replaces a faulty gene with a functional version.
- Example: Correcting mutations in diseases like SCID.
Editing Therapy
- Directly edits the DNA sequence using tools like CRISPR-Cas9, TALENs, or zinc-finger nucleases.
- Example: Correcting the HBB gene in sickle cell anemia.
Silencing Therapy
- Uses techniques like RNA interference (RNAi) or antisense oligonucleotides (ASOs) to turn off a harmful gene.
- Example: Silencing mutant huntingtin protein in Huntington’s disease.
Augmentation Therapy
- Introduces additional gene copies to increase its expression and enhance a specific function.
- Example: Enhancing immune response in cancer immunotherapy.
3. Based on Method of Delivery
In Vivo Gene Therapy
- Genes are delivered directly into the patient’s body.
- Example: Injecting viral vectors carrying therapeutic genes into target tissues.
Ex Vivo Gene Therapy
- Genes are introduced into cells outside the patient’s body, and the modified cells are reintroduced.
- Example: Modifying T-cells in CAR-T therapy for cancer.
4. Based on the Type of Vector Used
Viral Vectors
- Utilize modified viruses to deliver genetic material:
- Adenoviruses
- Adeno-associated viruses (AAV)
- Retroviruses
- Lentiviruses
- Example: Using AAV vectors for retinal gene therapy.
Non-Viral Vectors
- Employ chemical or physical methods to deliver genes:
- Liposomes
- Nanoparticles
- Electroporation
- Direct injection of naked DNA
5. Based on Treatment Intent
Curative Gene Therapy
- Aims to correct or cure the underlying genetic defect permanently.
- Example: Replacing defective SMN1 genes in spinal muscular atrophy (SMA).
Palliative Gene Therapy
- Focuses on managing symptoms or delaying disease progression rather than curing.
- Example: Delivering genes to produce proteins that reduce inflammation in arthritis.
6. Based on the Target Disease Type
Monogenic Disorders
- Targets diseases caused by mutations in a single gene.
- Examples: Hemophilia, and cystic fibrosis.
Polygenic Disorders
- Targets complex diseases influenced by multiple genes.
- Examples: Diabetes, heart disease.
Infectious Diseases
- Modifies immune cells or directly targets pathogens.
- Example: Gene editing for HIV resistance.
This classification provides a framework for understanding the diverse strategies and approaches in gene therapy, enabling tailored treatments for specific diseases.
The procedure of treatment with Gene therapy
Treating a patient with gene therapy typically involves several carefully planned and regulated steps. These steps ensure the safe and effective delivery of the therapeutic gene. Below is a general outline of the process:
First step: Disease Diagnosis and Patient Selection
- Genetic Testing: Identify the specific genetic mutation or defect causing the disease.
- Eligibility Assessment: Determine whether the patient is a suitable candidate for gene therapy based on:
- Type and severity of the disease.
- Availability of alternative treatments.
- Risks versus benefits.
- Informed Consent: Provide detailed information about the procedure, risks, and expected outcomes to the patient.
Second step: Therapeutic Gene Design
- Gene Identification: Select the therapeutic gene that can address the disease.
- This could involve adding, replacing, silencing, or editing the gene.
- Vector Development: Design a delivery system (vector) to carry the therapeutic gene into the target cells.
- Viral vectors (e.g., adenoviruses, lentiviruses, AAV).
- Non-viral methods (e.g., liposomes, nanoparticles, electroporation).
Third step: Preclinical Testing
- Conduct laboratory and animal studies to test:
- Safety.
- Efficacy.
- Optimal dosage.
- Delivery method.
Fourth step: Clinical Trials
- Phase I: Assess safety and determine the appropriate dose in a small group of patients.
- Phase II: Evaluate efficacy and monitor for side effects in a larger group.
- Phase III: Confirm effectiveness, monitor side effects, and compare with existing treatments.
Fifth step: Gene Delivery (Treatment Administration)
The method of delivering the therapeutic gene depends on the type of therapy:
In Vivo Gene Therapy
- The therapeutic gene is delivered directly into the patient’s body.
- Example: Injecting a viral vector carrying the gene into a specific tissue (e.g., retina, liver, or muscle).
Ex Vivo Gene Therapy
- Cell Harvesting involves extracting the patient’s cells (e.g., stem cells or immune cells) from blood or bone marrow.
- Gene Modification: Introduce the therapeutic gene into the cells in a controlled laboratory environment.
- Cell Expansion: Grow and multiply the modified cells.
- Reinfusion: Inject the modified cells back into the patient’s body.
Sixth step: Post-Treatment Monitoring
- Short-Term Monitoring: Assess immediate effects, such as immune reactions or side effects.
- Long-Term Follow-Up: Monitor the patient for potential delayed effects, such as:
- Sustained therapeutic benefit.
- Integration issues, like off-target effects in gene editing.
- Risk of cancer or other complications.
- Patients are often enrolled in registries to track long-term outcomes.
Seventh step: Evaluation of Outcomes
- Success is measured by improving the patient’s condition or correcting the genetic defect.
- Regular evaluations are conducted to monitor for relapse or disease progression.
Example Applications of Gene Therapy Procedures
- Spinal Muscular Atrophy (SMA): A one-time intravenous infusion of Zolgensma delivers a functional SMN1 gene to affected cells.
- Cancer (CAR-T Therapy): T-cells are extracted, modified to express cancer-targeting receptors, and reinfused into the patient.
- Hemophilia: A single injection of viral vectors carrying clotting factor genes helps restore the clotting process.
Challenges and Considerations
- Immune Response: The body might react to the vector or modified cells.
- Off-Target Effects: Unintended genetic modifications could cause side effects.
- Cost and Accessibility: Gene therapy can be expensive and may not be widely available.
- Ethical Issues: Concerns about germline editing and potential misuse.
Despite challenges, gene therapy is a promising and evolving field with the potential to cure many previously untreatable diseases.
Effectiveness of Gene therapy
The effectiveness of gene therapy varies depending on several factors, including the specific disease being treated, the type of therapy used, and individual patient characteristics. Here’s an overview of its effectiveness:
High Effectiveness for Monogenic Disorders
Gene therapy has shown remarkable success in treating diseases caused by mutations in a single gene, such as:
- Spinal Muscular Atrophy (SMA): Zolgensma, a one-time gene therapy, can dramatically improve motor function and survival rates in infants.
- Severe Combined Immunodeficiency (SCID): Often called “bubble boy disease,” gene therapy has successfully restored immune function in many patients.
- Hemophilia: Gene therapies like Hemgenix (for hemophilia B) have significantly reduced or eliminated the need for clotting factor infusions.
- Sickle Cell Anemia and Beta-Thalassemia: Gene editing techniques (e.g. CRISPR-based therapies) have cured some patients by correcting the defective genes in blood stem cells.
Promising Results in Cancer
Gene therapy has been transformative in oncology, particularly through CAR-T cell therapy:
- Leukemias and Lymphomas: CAR-T therapies (e.g. Kymriah, Yescarta) have achieved remission rates of up to 80% in some patients with resistant or relapsed cancers.
- Solid Tumors: Progress is slower, but ongoing research shows potential for personalized gene-based treatments.
Success in Ophthalmic Disorders
Gene therapies like Luxturna have restored partial vision in patients with Leber Congenital Amaurosis (LCA), a form of inherited blindness.
Variable Effectiveness in Polygenic or Complex Disorders
Diseases influenced by multiple genes or environmental factors are more challenging:
- Parkinson’s Disease: Gene therapies targeting dopamine production have shown improvement in symptoms but are not curative.
- Cardiovascular Diseases: Early trials in angiogenesis or heart failure show promise, but widespread clinical success is still under study.
Challenges Impacting Effectiveness
- Immune Response: The body’s immune system may attack the viral vectors used to deliver genes.
- Incomplete Delivery: Not all target cells may receive or express the therapeutic gene, reducing efficacy.
- Durability: Some therapies offer long-term relief, while others may require repeat treatments.
- Off-Target Effects: In gene editing, unintended changes to the DNA can limit effectiveness or cause side effects.
Success Rates
- Monogenic Disorders: Success rates are high, often achieving disease remission or substantial improvement (e.g. 90% effectiveness in SCID patients).
- Cancer (CAR-T Therapy): Remission rates range from 40% to 80%, depending on the type and stage of cancer.
- Vision Disorders: Luxturna has achieved significant vision restoration in a majority of patients.
- Rare Diseases: Many rare diseases have seen transformative results, although long-term studies are ongoing.
Overall Effectiveness
Gene therapy is highly effective for certain diseases, especially those caused by single-gene mutations. Its potential is growing for more complex conditions. However, the full extent of its effectiveness depends on overcoming challenges related to safety, delivery, and long-term outcomes.
In conclusion, while gene therapy is not yet universally effective for all conditions, it represents a powerful, often life-changing treatment for many diseases.
Drawbacks of Gene therapy
While it holds tremendous promise, it is not without its challenges and drawbacks. Here are some of the key limitations and risks associated with gene therapy:
Safety Concerns
- Immune Reactions: The body’s immune system may recognize the viral vectors used for gene delivery as foreign invaders, triggering an immune response that can cause inflammation, fever, or other complications.
- Toxicity: High doses of viral vectors may cause organ damage or systemic toxicity.
- Off-Target Effects: In gene-editing therapies (e.g. CRISPR), unintended genetic changes in non-target regions may lead to new health issues, including cancer.
Limited Efficacy
- Incomplete Delivery: Not all target cells may receive the therapeutic gene, reducing the overall effectiveness of the treatment.
- Transient Effects: Some therapies may not provide a permanent solution, requiring repeated treatments over time.
- Tissue Accessibility: Certain organs or tissues (e.g. brain, heart) can be difficult to target effectively with current delivery methods.
High Cost
- Gene therapies are among the most expensive treatments available. For example:
- Zolgensma (for SMA): Over $2 million per treatment.
- Luxturna (for inherited blindness): About $850,000 for both eyes.
- These high costs limit accessibility for many patients and place a significant burden on healthcare systems.
Ethical and Social Concerns
- Germline Editing: Changes to reproductive cells can be passed to future generations, raising ethical issues about unintended consequences and designer babies.
- Equity and Access: The high cost and complexity mean it may not be accessible to patients in low- and middle-income countries.
- Informed Consent: Patients may not fully understand the long-term risks and implications of experimental gene therapies.
Risk of Cancer
- In some cases, integrating the therapeutic gene into the host genome can disrupt normal gene function or activate oncogenes, leading to cancer. This was observed in early trials for SCID where some patients developed leukemia.
Long-Term Effects Unknown
- Durability of Treatment: It is unclear whether some gene therapies will provide permanent benefits or if their effects will diminish over time.
- Delayed Side Effects: The long-term safety of gene therapy, particularly for treatments involving viral vectors or genome editing, is still under study.
Complexity of Treatment
- Customized Therapies: Many gene therapies are tailored to individual patients, requiring highly specialized facilities, expertise, and equipment.
- Logistical Challenges: Ex vivo therapies (e.g. CAR-T cell therapy) involve multiple steps, including cell harvesting, genetic modification, and reinfusion, which can be time-consuming and technically demanding.
Technical Limitations
- Delivery Challenges: Efficiently and safely delivering therapeutic genes to the right cells and tissues remains a significant hurdle.
- Size of Genetic Material: Some genes are too large to fit into the most commonly used viral vectors.
- Gene Regulation: Ensuring the therapeutic gene is expressed at the right level and time can be challenging.
Psychological Impact
- Patients undergoing this therapy may experience emotional stress due to uncertainty about outcomes, risks of side effects, or high expectations for a cure.
Regulatory Hurdles
- Gene therapies often require extensive clinical testing to prove safety and efficacy, which can delay approval and availability.
Summary
Despite these drawbacks, it represents a revolutionary approach to treating previously untreatable diseases. Ongoing advancements in technology and research are addressing many of these challenges, making gene therapy safer, more effective, and accessible to a wider population.
Cost of Gene therapy in various countries
It represents a significant advancement in medical science, offering potential cures for various genetic disorders. However, the costs associated with these treatments are substantial and vary across different countries. Below is an overview of the costs for some notable gene therapies:
Zolgensma (Onasemnogene Abeparvovec)
- Indication: Spinal Muscular Atrophy (SMA)
- Cost:
- United States: Approximately $2.1 million per treatment.
- Europe: Prices may vary by country, often negotiated with healthcare systems.
Luxturna (Voretigene Neparvovec)
- Indication: Inherited Retinal Dystrophy
- Cost:
- United States: Around $850,000 for both eyes.
- Germany: Approximately €700,000 for both eyes.
Hemgenix (Etranacogene Dezaparvovec)
- Indication: Hemophilia B
- Cost:
- United States: Listed at $3.5 million per treatment.
- United Kingdom: Available through the NHS at £2.6 million per patient.
Kymriah (Tisagenlecleucel)
- Indication: Certain types of blood cancers
- Cost:
- United States: Approximately $475,000 per treatment.
- European Union: Prices vary; for example, in Germany, it’s around €320,000.
Libmeldy (Atidarsagene Autotemcel)
- Indication: Metachromatic Leukodystrophy
- Cost:
- European Union: Approximately €2.5 million per treatment.
Strimvelis
- Indication: Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)
- Cost:
- European Union: Around €594,000 per treatment.
Factors Influencing Cost Variations
- Healthcare System Negotiations: Prices can vary significantly based on negotiations between manufacturers and national healthcare systems or insurance providers.
- Regulatory Approvals: The availability and approval status differ by country, influencing accessibility and cost.
- Economic Factors: Currency exchange rates, local economic conditions, and healthcare funding models contribute to cost differences.
Considerations for Patients
- Insurance Coverage: In some countries, insurance may cover a portion of the treatment cost, but coverage varies widely.
- Financial Assistance Programs: Some pharmaceutical companies offer assistance programs to help patients afford treatment.
- Long-Term Value: While upfront costs are high, gene therapies may offer long-term savings by potentially curing conditions that require ongoing treatment.
It’s important to note that the field is rapidly evolving, and costs may change as new treatments are developed and existing therapies become more widely adopted. Patients considering gene therapy should consult with healthcare providers and financial advisors to understand the most current information and explore available options.
Conclusion
Gene therapy represents a groundbreaking advancement in medical science, offering hope for curing and managing diseases that were previously deemed untreatable. By addressing the root causes of genetic disorders, it has transformed the treatment landscape for conditions like spinal muscular atrophy, certain cancers, and inherited blindness. The technology has demonstrated remarkable success in correcting single-gene mutations, enhancing immune responses, and even paving the way for innovative approaches to complex diseases.
In summary, gene therapy is a revolutionary but evolving field. It holds immense potential to not only improve the quality of life for millions but also redefine the future of medicine by turning once-incurable conditions into treatable or even curable ones.
Frequently Asked Questions
1. What is gene therapy?
It is a medical technique that involves modifying or replacing faulty genes in a person’s cells to treat or prevent diseases. It aims to address the root cause of genetic disorders by introducing functional genes into the body.
2. How does gene therapy work?
It works by delivering therapeutic genes into a patient’s cells using vectors, such as viruses or non-viral methods. These vectors carry the gene to target cells, where it can replace, repair, or regulate faulty genes or provide new functions.
3. What diseases can be treated with gene therapy?
It has been used to treat several diseases, including:
- Spinal Muscular Atrophy (SMA)
- Severe Combined Immunodeficiency (SCID)
- Hemophilia
- Sickle Cell Anemia
- Certain cancers (e.g., leukemia and lymphoma)
- Inherited blindness (e.g., Leber Congenital Amaurosis)
- Metachromatic Leukodystrophy (MLD)
4. Is gene therapy a cure?
For some conditions, it offers a potential cure, such as SCID or SMA. However, in other cases, it may only provide symptom relief or delay disease progression. The effectiveness depends on the disease and the type of therapy.
5. Is gene therapy safe?
It is considered safe when properly regulated, but it carries risks like immune reactions, off-target effects, or potential toxicity. Rigorous clinical trials and monitoring aim to minimize these risks.
6. How much does gene therapy cost?
It is expensive due to its complexity. Costs can range from hundreds of thousands to several million dollars per treatment. For example:
- Zolgensma (SMA): $2.1 million.
- Luxturna (blindness): $850,000. Prices vary by country and availability of insurance or government subsidies.
7. What are the benefits of gene therapy?
It offers several benefits:
- Treats the root cause of genetic diseases.
- Reduces the need for lifelong medications or treatments.
- Provides potential cures for previously untreatable conditions.
- Improves the quality of life for patients with severe disorders.
8. What are the risks of gene therapy?
Some risks include:
- Immune system reactions to the vector.
- Off-target effects that may disrupt other genes.
- Long-term side effects that are not yet fully understood.
- Possibility of cancer due to gene insertion in the wrong location.
9. Is gene therapy available for everyone?
It is not yet widely available due to high costs, limited infrastructure, and the need for specialized expertise. It is typically offered in developed countries and major research institutions, but access is expanding as technology advances.
10. What does the future hold for gene therapy?
The future is promising, with ongoing research aimed at:
- Making treatments safer and more affordable.
- Developing therapies for complex diseases like diabetes and neurodegenerative disorders.
- Expanding accessibility to underserved populations.
- Refining gene-editing technologies like CRISPR for precision treatments.
Gene therapy continues to revolutionize medicine, offering hope and transformative outcomes for patients worldwide.
Reference: https://en.wikipedia.org/wiki/Gene_therapy
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