Towards a rAAV-based gene therapy for ADA-SCID: From ADA deficiency to current and future treatment strategies

Pharmacogenomics

Volumn 9, Issue 7, 2008, Pages 947-968

  Silver, Jared N ^a   Flotte, Terence R ^b  

^a UNIVERSITY OF FLORIDA COLLEGE OF MEDICINE   (United States)

^b UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL   (United States)

Author keywords

ADA SCID; Adenosine deaminase; BMT; Gene therapy; Immune deficiency; PEG ADA; rAAV; Retroviral vectors

Indexed keywords

ADENOSINE DEAMINASE; ADENOVIRUS VECTOR; ALPHA 1 ANTITRYPSIN; BETA GLUCURONIDASE; BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 9; BUSULFAN; GRANULOCYTE COLONY STIMULATING FACTOR; HERPESVIRUS VECTOR; IMMUNOGLOBULIN; LENTIVIRUS VECTOR; MELPHALAN; PARVOVIRUS VECTOR; PEGADEMASE; RETROVIRUS VECTOR; TRANSMEMBRANE CONDUCTANCE REGULATOR; RECOMBINANT PROTEIN;

ADENOSINE DEAMINASE DEFICIENCY; ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; ALPHA 1 ANTITRYPSIN DEFICIENCY; AUTOIMMUNE DISEASE; AUTOLOGOUS PERIPHERAL BLOOD STEM CELL TRANSPLANTATION; BONE MARROW TRANSPLANTATION; CLINICAL FEATURE; CLINICAL TRIAL; CYSTIC FIBROSIS; DRUG COST; DRUG EFFICACY; DRUG MECHANISM; DRUG POTENCY; DRUG SAFETY; DRUG TOLERABILITY; ENGRAFTMENT; ENZYME REPLACEMENT; EOSINOPHILIA; EX VIVO GENE TRANSFER; FATALITY; GENE EXPRESSION REGULATION; GRAFT VERSUS HOST REACTION; HEALTH CARE ACCESS; HEMOLYTIC ANEMIA; HEMOPHILIA A; HEMOPHILIA B; HUMAN; HYPERGAMMAGLOBULINEMIA; IMMUNE DYSREGULATION; IN VIVO GENE TRANSFER; INFECTIOUS COMPLICATION; MUCOPOLYSACCHARIDOSIS TYPE 7; MYELOABLATIVE CONDITIONING; NONHUMAN; OPPORTUNISTIC INFECTION; PATIENT COMPLIANCE; PHENOTYPE; PROGNOSIS; PULMONARY HYPERTENSION; REPEATED DRUG DOSE; REVIEW; SINGLE DRUG DOSE; STEM CELL MOBILIZATION; TREATMENT FAILURE; TREATMENT OUTCOME; UNSPECIFIED SIDE EFFECT; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VIRUS INFECTION; VIRUS RECOMBINANT; ADENO ASSOCIATED VIRUS; ENZYMOLOGY; GENE THERAPY; GENE VECTOR; GENETICS; METHODOLOGY; SEVERE COMBINED IMMUNODEFICIENCY;

ADENO-ASSOCIATED VIRUS;

ADENOSINE DEAMINASE; DEPENDOVIRUS; GENE THERAPY; GENETIC VECTORS; HUMANS; RECOMBINANT PROTEINS; SEVERE COMBINED IMMUNODEFICIENCY;

EID: 49949091947     PISSN: 14622416     EISSN: 17448042     Source Type: Journal    
DOI: 10.2217/14622416.9.7.947     Document Type: Review

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