gene therapy

What is gene therapy?

A medical approach that modifies a patient's genetic material to treat or prevent diseases by correcting, replacing, or altering defective genes

key challenges of gene therapy

• Delivery Mechanisms: Efficient delivery into target cells is challenging.

• Safety Concerns: Risk of immune reactions, inflammation, or insertional mutagenesis.

• Durability: Short-lived effects in dividing cells.

• Cost: High production and delivery costs.

• Ethical Issues: Concerns about germline modifications.

• Scalability: Difficulty in producing large quantities of vectors

process of gene therapy

1. Identification of the target gene.

2. Selection of delivery methods (e.g., viral or non-viral vectors).

3. Design of therapeutic genes for proper expression and safety.

4. Targeting specific tissues for delivery.

5. Strategies to evade immune responses.

6. Preclinical testing and clinical trials for validation.

What is Zolgensma, and what does it treat?

• Disease: Spinal Muscular Atrophy (SMA).

• Mechanism: Delivers a functional copy of the SMN1 gene via a viral vector to restore SMN protein production.

• Impact: Dramatically improves survival and quality of life in SMA patients

What are the safety concerns in gene therapy?

• Immune responses or inflammation.

• Risk of insertional mutagenesis leading to cancer.

• Unintended effects on non-target cells

What is the significance of preclinical testing in gene therapy?

Ensures efficacy, safety, and proper expression of therapeutic genes before human trials