Gene Therapy: Gene Delivery Systems And Treatment Of Rare Diseases

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The book delves into the definition and regulatory framework of gene therapy as outlined by the European Medicines Agency, highlighting its unique characteristics involving recombinant nucleic acids. It emphasizes the distinction between gene therapy and vaccines, noting that the latter is not permitted within this category. The narrative also reflects on the clinical successes achieved between 2009 and 2011, showcasing advancements in treating conditions such as Leber congenital amaurosis, X-linked SCID, ADA SCID, adrenoleukodystrophy, and Parkinson's disease, fostering renewed optimism in the field.