Gene Therapy
By targeting and correcting faulty genes, gene therapy offers a transformative approach to treating hereditary and rare genetic disorders. This innovative therapy unlocks solutions previously thought impossible, giving patients a brighter, healthier future.
The Science Behind Gene Therapy
Gene therapy works by introducing genetic material into a patient’s cells to correct or replace defective genes. This process can involve several methods, such as using viral vectors to deliver healthy genes or CRISPR-based gene editing to repair specific mutations.
With advancements in precision medicine, gene therapy can treat a variety of hereditary and rare genetic disorders by targeting the source of the condition rather than just managing symptoms. This therapy not only halts disease progression but can also restore normal cell function.
Key Benefits of Gene Therapy
Addresses Root Causes
Repairs faulty genes to tackle diseases at their source.
Long-term Relief
Provides lasting solutions that reduce or eliminate disease symptoms.
Personalised Treatment
Tailored to each individual’s unique genetic profile for precise outcomes.
Improved Quality of Life
Enables patients to lead healthier and more independent lives.
Why Choose JCRC-ABT CellGene Innovations?
Our approach to gene therapy is grounded in rigorous science, ethical practices, and personalized care. From understanding your unique genetic profile to providing a treatment plan that fits your needs, we focus on delivering safe, effective, and meaningful results. With a team of experienced professionals and a patient-first philosophy, we ensure you feel supported at every stage of your journey.
1. What conditions can gene therapy treat?
Gene therapy is effective for hereditary conditions like sickle cell anemia, certain cancers, and rare genetic disorders caused by specific gene mutations.
2. How does gene therapy work?
The therapy uses specially engineered vectors (often harmless viruses) to deliver healthy genetic material into your cells, replacing or repairing defective genes.
3. Is gene therapy safe?
Yes. Gene therapy is conducted under strict safety protocols and adheres to international standards. At JCRC-ABT, we partner with global leaders to ensure every procedure is safe and effective.
4. Is gene therapy a one-time treatment?
In many cases, gene therapy is designed to provide long-term or even permanent benefits, often requiring only a single treatment.
5. How long does it take to see results?
Results depend on the condition and the patient, but many see significant improvements within months of treatment.
CASE STUDY
Case Studies on Gene Therapy
Leber’s Congenital Amaurosis (LCA)
The gene therapy Luxturna restored partial vision in patients with RPE65 gene mutations. Clinical trials demonstrated improved light sensitivity and navigation abilities, with effects persisting for over three years.
Sickle Cell Disease
A study in The New England Journal of Medicine highlighted the success of CRISPR-based gene editing in correcting the HBB gene mutation. Treated patients experienced a significant reduction in vaso-occlusive crises and showed normalized hemoglobin levels within months.
Spinal Muscular Atrophy (SMA)
The FDA-approved gene therapy Zolgensma demonstrated remarkable efficacy in infants with SMA Type 1. A single intravenous dose restored the SMN1 gene, leading to improved motor function and survival rates, with many patients achieving milestones like sitting unassisted.
Cystic Fibrosis
A trial targeting CFTR gene mutations delivered via aerosolized vectors showed improved pulmonary function in patients with cystic fibrosis. Published in The Lancet Respiratory Medicine, the therapy reduced hospitalizations and improved quality of life metrics over 12 months.
Hemophilia B
Gene therapy using an AAV vector to deliver the FIX gene resulted in sustained Factor IX production in patients. A landmark study in Blood reported a significant reduction in bleeding episodes and factor replacement therapy use, lasting up to 5 years.
