Gene and cell therapy for children - New medicines, new challenges?

Advanced Drug Delivery Reviews

Volumn 73, Issue , 2014, Pages 162-169

  Buckland, Karen F ^a,c   Bobby Gaspar, H ^a,b  

^a UNIVERSITY COLLEGE LONDON   (United Kingdom)

^b Department of Clinical Immunology Great Ormond Street Hospital NHS Foundation Trust ^*  (United Kingdom)

^c Cellular Therapies Great Ormond Street Hospital NHS Foundation Trust   (United Kingdom)

Author keywords

ATMP; Autologous; Biologics; Cell therapy; Gene therapy; GMP; Stem cell; Translational; Vector

Indexed keywords

CELL CULTURE; DISEASE CONTROL; DISEASES; EXPERIMENTS; GENETIC ENGINEERING; NEUROLOGY; PATIENT TREATMENT; PEDIATRICS; REPAIR; STEM CELLS; VECTORS; CELL ENGINEERING; CELLS; COST EFFECTIVENESS; CYTOLOGY; GENES; PATIENT REHABILITATION;

ATMP; AUTOLOGOUS; BIOLOGICS; CELL THERAPY; GMP; TRANSLATIONAL;

GENE THERAPY;

AGENTS ACTING ON THE EYE; ALIPOGENE TIPARVOVEC; RETROVIRUS VECTOR; RPE65 PROTEIN; UNCLASSIFIED DRUG; ADENOSINE DEAMINASE; ANTISENSE OLIGONUCLEOTIDE; BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 9; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; PARVOVIRUS VECTOR; PIGMENT EPITHELIUM DERIVED FACTOR; SMALL INTERFERING RNA; DRUG;

ADENOVIRUS; ADRENOLEUKODYSTROPHY; BLINDNESS; CELL FUNCTION; CELL THERAPY; CHILD; CHILDHOOD DISEASE; CLINICAL EFFECTIVENESS; CORNEA DISEASE; COST BENEFIT ANALYSIS; COST EFFECTIVENESS ANALYSIS; DRUG EFFICACY; EX VIVO GENE TRANSFER; EX VIVO STUDY; GENE; GENE SILENCING; GENE THERAPY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMOPHILIA; HUMAN; HYPERLIPOPROTEINEMIA TYPE 1; IN VIVO GENE TRANSFER; IN VIVO STUDY; METABOLIC DISORDER; METACHROMATIC LEUKODYSTROPHY; MOLECULAR DYNAMICS; MUCOPOLYSACCHARIDOSIS; MUSCLE REGENERATION; MUSCULAR DYSTROPHY; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; PANCREAS ISLET CELL; PRIORITY JOURNAL; RESPIRATORY TRACT DISEASE; RETINA DISEASE; RETROVIRUS; REVIEW; RNA INTERFERENCE; RPE65 GENE; SIGNAL TRANSDUCTION; SKIN CELL; T LYMPHOCYTE; TREATMENT RESPONSE; ADENOSINE DEAMINASE DEFICIENCY; BONE MARROW TRANSPLANTATION; CANCER CELL; CANCER SURVIVAL; CELL CULTURE; CELL KILLING; CHILD HEALTH CARE; CHILDHOOD CANCER; CLINICAL TRIAL (TOPIC); CONVALESCENCE; CYSTIC FIBROSIS; DUCHENNE MUSCULAR DYSTROPHY; EPIDERMOLYSIS BULLOSA; EPILEPSY; GENE DELIVERY SYSTEM; GENETIC ENGINEERING; GENETIC TRANSDUCTION; HEALTH CARE INDUSTRY; HEMOPHILIA A; HEMOPHILIA B; IMMUNE DEFICIENCY; INSULIN DEPENDENT DIABETES MELLITUS; LEBER CONGENITAL AMAUROSIS; LEUKODYSTROPHY; LYMPHOCYTE SUBPOPULATION; LYMPHOCYTE TRANSFER; MEDICAL RESEARCH; MELANOMA; METHODOLOGY; MUSCLE DISEASE; PANCREAS ISLET TRANSPLANTATION; PATIENT SAFETY; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); RETINITIS PIGMENTOSA; SOMATIC CELL THERAPY; STEM CELL TRANSPLANTATION; TARGET CELL; TECHNOLOGY; TUMOR ASSOCIATED LEUKOCYTE; ANIMAL; BIOLOGICAL THERAPY; PEDIATRICS;

ANIMALS; CELL- AND TISSUE-BASED THERAPY; CHILD; GENETIC THERAPY; HUMANS; PEDIATRICS; PHARMACEUTICAL PREPARATIONS;

EID: 84902316060     PISSN: 0169409X     EISSN: 18728294     Source Type: Journal    
DOI: 10.1016/j.addr.2014.02.010     Document Type: Review

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