Vectors for the treatment of autoimmune disease

Gould, David; Favorov, Peter

doi:10.1038/sj.gt.3302018

Cited by 37 publications

(15 citation statements)

References 201 publications

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“…The central goal of AAV gene therapy for these conditions is to shift the balance to a non-inflammatory phenotype by expressing transgenes to impart tolerance to the self-antigen or antagonize the inflammatory cytokines (Evans et al 2004;Gould and Favorov 2003;Tarner and Fathman 2002). These strategies have utilized systemic expression of therapy by intramuscular administration of AAV vectors or the delivery of vector directly to the affected tissue.…”

Section: Gene Therapy For Obesitymentioning

confidence: 99%

Treatment of human disease by adeno-associated viral gene transfer

2006

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During the past decade, in vivo administration of viral gene transfer vectors for treatment of numerous human diseases has been brought from bench to bedside in the form of clinical trials, mostly aimed at establishing the safety of the protocol. In preclinical studies in animal models of human disease, adeno-associated viral (AAV) vectors have emerged as a favored gene transfer system for this approach. These vectors are derived from a replication-deficient, non-pathogenic parvovirus with a single-stranded DNA genome. Efficient gene transfer to numerous target cells and tissues has been described. AAV is particularly efficient in transduction of non-dividing cells, and the vector genome persists predominantly in episomal forms. Substantial correction, and in some instances complete cure, of genetic disease has been obtained in animal models of hemophilia, lysosomal storage disorders, retinal diseases, disorders of the central nervous system, and other diseases. Therapeutic expression often lasted for months to years. Treatments of genetic disorders, cancer, and other acquired diseases are summarized in this review. Vector development, results in animals, early clinical experience, as well as potential hurdles and challenges are discussed.

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Section: Gene Therapy For Obesitymentioning

confidence: 99%

Treatment of human disease by adeno-associated viral gene transfer

2006

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“…Selection of a vector to mediate gene transfer for the treatment of autoimmune diseases can involve multiple considerations [11], including target tissue, length of expression desired, and size of the transgene cassette. We began to explore the use of serotype 2 rAAV (rAAV2) vectors for salivary gland gene transfer in large part due to the potent rAd5 vector associated immune response, in addition to our interest in developing novel and useful therapies for Sjogren's [22] a Abbreviations used in this table are as follows: interleukin-10, IL-10; viral interleukin-10, vIL-10; interleukin-4, IL-4; CTLA-4Ig, soluble form of cytotoxic T lymphocyte-associated antigen 4; sTNFR1, soluble form of the tumor necrosis factor receptor 1.…”

Section: Vector Selection and Deliverymentioning

confidence: 99%

Advances in vector-mediated gene transfer

et al. 2003

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“…For example, some vectors can be immunogenic and cause inflammation, while others may induce malignant transformation of the target cell or may reassume the competence for replication. [36] This heterogeneity must be factored into estimates of risks associated with gene therapy. The following additional factors should be taken into account: the nature of the transgene, which may modify host responses; the nature of the target cell; distribution of the vector in the body; and the nature of the disease.…”

Section: Safety Of Viral Vectorsmentioning

confidence: 99%