Question

Discuss in-utero enzyme replacement therapy and gene therapy - Enlist success and challenges for each.

Hint:

Both in-utero enzyme replacement therapy and gene therapy hold significant potential, but they face challenges related to delivery methods, safety, and long-term effectiveness.

Answer 1

In-utero enzyme replacement therapy (ERT) and gene therapy are advanced treatment options for genetic disorders. Both therapies aim to address the underlying genetic causes, but they differ in their approach. In-Utero Enzyme Replacement Therapy (ERT):
In-utero ERT involves the administration of enzymes to a fetus in utero to compensate for the lack of functional enzymes due to genetic disorders like lysosomal storage diseases. Successes:
1. Early Intervention: ERT administered early can prevent the progression of disorders, reducing the severity of symptoms post-birth.
2. Improved Outcomes: Studies have shown that ERT can lead to better developmental outcomes and organ function in treated infants. Challenges:
1. Delivery Method: The challenge of safely delivering enzymes across the placenta without causing harm to the fetus.
2. Limited Availability: The therapy is still in the experimental phase for many diseases, with limited clinical availability and regulatory approval. Gene Therapy:
Gene therapy involves introducing, removing, or altering genetic material within a patient's cells to treat or prevent disease. Successes:
1. Long-Term Effectiveness: Gene therapy has shown promise in providing long-lasting effects by correcting the genetic defect at the source.
2. Success in Rare Diseases: Gene therapy has had notable success in treating rare genetic diseases such as spinal muscular atrophy and certain types of inherited blindness. Challenges:
1. Immune Response: One major challenge is the immune system's potential rejection of the modified cells or viral vectors used in gene delivery.
2. Safety Concerns: There are concerns about unintended genetic changes or insertional mutagenesis, which could lead to cancer or other health problems.