Genetic medicines for CF: Hype versus reality

Pediatric Pulmonology

Volumn 51, Issue , 2016, Pages S5-S17

  Alton, Eric W F W ^a   Boyd, A Christopher ^a   Davies, Jane C ^a   Gill, Deborah R ^a   Griesenbach, Uta ^a   Harrison, Patrick T ^b   Henig, Noreen ^c   Higgins, Tracy ^a   Hyde, Stephen C ^a   Innes, J Alastair ^a   Korman, Michael S D ^d  

^a UK Cystic Fibrosis Gene Therapy Consortium   (United Kingdom)

^b UNIVERSITY COLLEGE CORK   (Ireland)

^c PROQR THERAPEUTICS   (Netherlands)

^d UNIVERSITY OF TÜBINGEN   (Germany)

Author keywords

cystic fibrosis (CF); gene therapy; genome editing; lung

Indexed keywords

COMPLEMENTARY DNA; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; LENTIVIRUS VECTOR; MESSENGER RNA; OLIGONUCLEOTIDE; PLASMID DNA; VIRAL PROTEIN;

ADENO ASSOCIATED VIRUS; ADENOVIRIDAE; BACULOVIRIDAE; CFTR GENE; CYSTIC FIBROSIS; DISEASE COURSE; DRUG EFFICACY; DRUG SAFETY; DRUG TOLERABILITY; GENE; GENE DELIVERY SYSTEM; GENE EXPRESSION; GENE TRANSFER; GENETIC TRANSCRIPTION; HUMAN; MOLECULAR CLONING; NONHUMAN; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); PRIORITY JOURNAL; REVIEW; RNA REPAIR; VIRAL GENE THERAPY;

EID: 84988474031     PISSN: 87556863     EISSN: 10990496     Source Type: Journal    
DOI: 10.1002/ppul.23543     Document Type: Review

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