Adeno-Associated Virus (AAV) is a non-enveloped virus that can be engineered to deliver DNA to target cells and has become a major focus in gene therapy. AAV is a versatile viral vector that can be tailored for specific gene therapy applications. It is safe and effective in preclinical and clinical settings and can be used in a wide range of clinical applications for various diseases due to its unique biological and biophysical properties. Recombinant AAV (rAAV) is a protein-based nanoparticle that can deliver DNA to the nucleus of a cell. It is used in gene therapy because it does not integrate into the host genome and can be safely used in clinical settings. The design of rAAV vectors involves considerations such as the size of the expression cassette, the choice of promoters, and the use of tissue-specific promoters. AAV capsid selection and optimization are important for targeting specific cell and tissue types. AAV has been shown to be less immunogenic than other viruses, but pre-existing immunity to AAV can affect its clinical efficacy. AAV is produced in living systems and requires careful manufacturing and purification processes. AAV has been used in various clinical trials for different diseases, including inherited retinal diseases, hemophilia, and muscular dystrophy. The delivery strategies for AAV include systemic, intramuscular, and CNS delivery. AAV has shown promise in treating various diseases, and ongoing research is focused on optimizing its use in gene therapy. The future of AAV as a gene therapy vector is promising, with continued efforts to improve its safety, efficacy, and clinical applications.Adeno-Associated Virus (AAV) is a non-enveloped virus that can be engineered to deliver DNA to target cells and has become a major focus in gene therapy. AAV is a versatile viral vector that can be tailored for specific gene therapy applications. It is safe and effective in preclinical and clinical settings and can be used in a wide range of clinical applications for various diseases due to its unique biological and biophysical properties. Recombinant AAV (rAAV) is a protein-based nanoparticle that can deliver DNA to the nucleus of a cell. It is used in gene therapy because it does not integrate into the host genome and can be safely used in clinical settings. The design of rAAV vectors involves considerations such as the size of the expression cassette, the choice of promoters, and the use of tissue-specific promoters. AAV capsid selection and optimization are important for targeting specific cell and tissue types. AAV has been shown to be less immunogenic than other viruses, but pre-existing immunity to AAV can affect its clinical efficacy. AAV is produced in living systems and requires careful manufacturing and purification processes. AAV has been used in various clinical trials for different diseases, including inherited retinal diseases, hemophilia, and muscular dystrophy. The delivery strategies for AAV include systemic, intramuscular, and CNS delivery. AAV has shown promise in treating various diseases, and ongoing research is focused on optimizing its use in gene therapy. The future of AAV as a gene therapy vector is promising, with continued efforts to improve its safety, efficacy, and clinical applications.