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8 papers analyzed
These studies suggest that the challenges of developing gene therapies for genetic disorders include delivery and duration of expression, overcoming biological barriers, large-scale manufacturing, precision of gene delivery, and addressing safety, efficiency, and societal concerns.
20 papers analyzed
Gene therapy holds significant promise for treating genetic disorders by directly addressing the underlying genetic causes. Despite its potential, the development and implementation of gene therapies face numerous challenges that need to be addressed to ensure their safety, efficacy, and scalability.
Vector Design and Delivery Challenges:
Immune Response and Safety Concerns:
Scalability and Manufacturing:
Specific Challenges in Neurological Disorders:
Technological and Methodological Advances:
Clinical Trial Setbacks and Progress:
Developing gene therapies for genetic disorders involves overcoming significant challenges related to vector design, immune response management, scalability, and specific disease-related hurdles. While technological advancements offer new possibilities, ensuring the safety, efficacy, and cost-effectiveness of these therapies remains a critical focus for future research and development.
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