Gene, stem cell, and future therapies for orphan diseases

Clinical Pharmacology and Therapeutics

Volumn 92, Issue 2, 2012, Pages 182-192

  Phillips, M I ^a  

^a KECK GRADUATE INSTITUTE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ABC TRANSPORTER; ABC TRANSPORTER A4; ADENOVIRUS VECTOR; ALPHA 1 ANTITRYPSIN; AMINODEAMINASE; ANGIOSTATIN PLUS ENDOSTATIN; BB 1608; CANCER VACCINE; CYTOSINE DEAMINASE; DEAMINASE; DOCETAXEL; DYSTROPHIN; ET 101; ETEPLIRSEN; FANCONI ANEMIA GROUP C PROTEIN; FLUCYTOSINE; ICT 107; INTERLEUKIN 2; LENTIVIRUS VECTOR; MONOCLONAL ANTIBODY; NOTCH PROTEIN MONOCLONAL ANTIBODY; ORNITHINE CARBAMOYLTRANSFERASE; PLERIXAFOR; RECOMBINANT ADENO ASSOCIATED VIRUS VECTOR 2; RETINA PIGMENT EPITHELIUM 65 KDA; RETROVIRUS VECTOR; SL 401; STARGEN; UNCLASSIFIED DRUG; UNINDEXED DRUG; VERASTEM; VIRUS VECTOR; VISUAL PIGMENT; WISKOTT ALDRICH SYNDROME PROTEIN;

ABSENCE OF SIDE EFFECTS; ADENO ASSOCIATED VIRUS; ADRENOLEUKODYSTROPHY; ALPHA 1 ANTITRYPSIN DEFICIENCY; ARTICLE; BONE MARROW TRANSPLANTATION; BRAIN DEATH; CANCER STEM CELL; CARCINOGENESIS; DISEASE MODEL; DNA MODIFICATION; DNA REPAIR; DNA SEQUENCE; DRUG EFFICACY; DRUG MEGADOSE; DRUG SAFETY; DUCHENNE MUSCULAR DYSTROPHY; ELECTROPORATION; EMBRYONIC STEM CELL; FANCONI ANEMIA; GENE MUTATION; GENE OVEREXPRESSION; GENE SILENCING; GENE SWITCHING; GENE THERAPY; GENETIC ENGINEERING; GENETIC RECOMBINATION; GLIOBLASTOMA; GLIOMA; HEMOPHILIA; HUMAN; LEBER CONGENITAL AMAUROSIS; LENTIVIRINAE; LEUKEMIA; LOW DRUG DOSE; MESENCHYMAL STEM CELL TRANSPLANTATION; METASTATIC MELANOMA; MULTIPLE ORGAN FAILURE; MUTAGENESIS; NEURAL STEM CELL TRANSPLANTATION; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; ORNITHINE TRANSCARBAMYLASE DEFICIENCY; PARACRINE SIGNALING; PLURIPOTENT STEM CELL; PRIORITY JOURNAL; PROMOTER REGION; RETINA MACULA AGE RELATED DEGENERATION; RETINITIS PIGMENTOSA; RETROVIRUS; SIDE EFFECT; SPINAL CORD INJURY; STARGARDT DISEASE; STEM CELL TRANSPLANTATION; VIRAL GENE DELIVERY SYSTEM; WISKOTT ALDRICH SYNDROME; X LINKED SEVERE COMBINED IMMUNODEFICIENCY;

CLINICAL TRIALS AS TOPIC; DNA REPAIR; GENE TARGETING; GENE THERAPY; HUMANS; RARE DISEASES; STEM CELL TRANSPLANTATION;

EID: 84864153400     PISSN: 00099236     EISSN: 15326535     Source Type: Journal    
DOI: 10.1038/clpt.2012.82     Document Type: Article

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