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Adeno-associated virus therapies: Pioneering solutions for human genetic diseases

19 Sept 2024

Abstract excerpt

Adeno-associated virus (AAV) has emerged as a fundamental component in the gene therapy landscape, widely acknowledged for its effectiveness in therapeutic gene delivery. The success of AAV-based therapies, such as Luxturna and Zolgensma, underscores their potential as a leading vector in gene therapy. This article provides an in-depth review of the development and mechanisms of AAV vector-based therapies,...

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CRISPR and Genetic Engineering
Viral Infections and Immunology Research
Virus-based gene therapy research

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Identifiers

PMID: 39322487
DOI: 10.1016/j.cytogfr.2024.09.003

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 1 Sept 2026.

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