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  2. Adeno-associated virus - Wikipedia

    en.wikipedia.org/wiki/Adeno-associated_virus

    A number of candidate T cell stimulating epitopes have been identified within the AAV capsid protein VP1, which may be attractive targets for modification of the capsid if the virus is to be used as a vector for gene therapy. [107] [108]

  3. Virotherapy - Wikipedia

    en.wikipedia.org/wiki/Virotherapy

    Vectors made from Adeno-associated virus are one of the most established products used in clinical trials today. It was initially attractive for the use of gene therapy due to it not being known to cause any disease along with several other features. [27] It has also been engineered so that it does not replicate after the delivery of the gene. [27]

  4. Gene therapy of the human retina - Wikipedia

    en.wikipedia.org/wiki/Gene_therapy_of_the_human...

    Development continued, and in December 2017 the FDA approved Voretigene neparvovec (Luxturna), an adeno-associated virus vector-based gene therapy for children and adults with biallelic RPE65 gene mutations responsible for retinal dystrophy, including Leber congenital amaurosis. People must have viable retinal cells as a prerequisite for the ...

  5. Self-complementary adeno-associated virus - Wikipedia

    en.wikipedia.org/wiki/Self-Complementary_Adeno...

    Self-complementary adeno-associated virus (scAAV) is a viral vector engineered from the naturally occurring adeno-associated virus (AAV) to be used as a tool for gene therapy. [1] Use of recombinant AAV (rAAV) has been successful in clinical trials addressing a variety of diseases. [ 2 ]

  6. Viral vector - Wikipedia

    en.wikipedia.org/wiki/Viral_vector

    Adeno Associated Virus (AAV) (lower panel) – To produce AAV, package a gene of interest into the AAV-ITR vector and transfect cells with a Helper vector and the Rep/Cap DNA integration vector. Adeno-associated viruses (AAVs) are relatively small single-stranded DNA viruses belonging to Parvoviridae and, like lentiviral vectors, AAVs can ...

  7. Gene therapy - Wikipedia

    en.wikipedia.org/wiki/Gene_therapy

    A number of viruses have been used for human gene therapy, including viruses such as lentivirus, adenoviruses, herpes simplex, vaccinia, and adeno-associated virus. [5] Adenovirus viral vectors (Ad) temporarily modify a cell's genetic expression with genetic material that is not integrated into the host cell's DNA.

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