Module232025

Gene Therapy for Pediatrics : Key Points

Gene Therapy Overview :

Uses recombinant nucleic acids to treat diseases through ex vivo , in vivo , or in situ strategies.

Advances in Viral Vectors :

Improved viral vectors, like lentiviruses, enable rare disorder treatments despite challenges like immunogenicity and production issues. Non-Viral Vectors :

Promising carriers (e.g., PEI, dendrimers) offer safer alternatives but struggle with toxicity and stability.

CRISPR/Cas9 Editing :

Precisely targets genetic diseases like β - thalassemia and “butterfly child” syndrome.

EMA Guidelines for GTMPs :

Ensure quality, safety, and efficacy with specific vector evaluations.

Challenges :

High costs, limited shelf life, and need for long-term monitoring restrict broader applications.

Quality (CMC) Development Considerations

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