Therapeutic possibilities in muscular dystrophy: The hope versus the hype

Neuromuscular Disorders

Volumn 12, Issue 2, 2002, Pages 113-116

  Dubowitz, V ^a  

^a IMPERIAL COLLEGE SCHOOL OF MEDICINE   (United Kingdom)

Author keywords

Cell therapy; Duchenne; Gene therapy; Muscular dystrophy; Protein upregulation; Therapy

Indexed keywords

AMINOGLYCOSIDE ANTIBIOTIC AGENT; DNA; DYSTROPHIN; GENTAMICIN; PROTEIN; UTROPHIN; VIRUS VECTOR;

BICEPS BRACHII MUSCLE; BONE MARROW CELL; CELL CULTURE; CELL DEATH; CELL MIGRATION; CELL TRANSFER; CLINICAL TRIAL; CONFERENCE PAPER; DISEASE MODEL; DUCHENNE MUSCULAR DYSTROPHY; GENE DELETION; GENE THERAPY; HUMAN; LIMB GIRDLE MUSCULAR DYSTROPHY; MOLECULAR GENETICS; MUSCLE ATROPHY; MUSCLE STRENGTH; MYOBLAST; NOMENCLATURE; NONHUMAN; PHENOTYPE; PRIORITY JOURNAL; PROTEIN EXPRESSION; SEPTICEMIA; STOP CODON; TREATMENT FAILURE; TREATMENT OUTCOME;

ANIMALS; DISEASE MODELS, ANIMAL; HUMANS; MUSCULAR DYSTROPHIES; MUSCULAR DYSTROPHY, ANIMAL; RESEARCH DESIGN;

EID: 0036133504     PISSN: 09608966     EISSN: None     Source Type: Journal    
DOI: 10.1016/S0960-8966(01)00319-4     Document Type: Conference Paper

References (22)

1. Muscle transplantation and regeneration in the dystrophic hamster
2. Muscle transplantation between normal and dystrophic mice
3. Isoenzyme studies in the identification of transplanted muscle in the mouse
4. Evidence of fusion between host and donor myoblasts in skeletal muscle grafts
5. Use of mononuclear precursor cells to insert allogeneic genes into growing mouse muscles
6. Partial correction of an inherited biochemical defect of skeletal muscle by grafts of normal muscle precursor cells
7. Conversion of mdx myofibres from dystrophin-negative to positive by injection of normal myoblasts
8. Dystrophin is expressed in mdx skeletal muscle fibres after normal myoblast implantation
9. Myoblast transfer therapy
10. Myoblast transfer in Duchenne muscular dystrophy
11. Normal dystrophin transcripts detected in Duchenne muscular dystrophy patients after myoblast transplantation
12. Dystrophin expression in the mdx mouse restored by stem cell transplantation
13. Successful myoblast transplantation in primates depends on appropriate cell delivery and induction of regeneration in the host muscle
14. Myoblast transplantation in whole muscle of nonhuman primates
15. Human dystrophin expression in mdx mice after intramuscular injection of DNA constructs
16. Gene therapy: Present situation and future prospects
17. Human dystrophin expression corrects the myopathic phenotype in transgenic mdx mice
18. Efficient adenovirus-mediated transfer of a human minidystrophin gene to skeletal muscle of mdx mice
19. Amelioration of the dystrophic phenotype of mdx mice using truncated utrophin transgene
20. Aminoglycoside antibiotics restore dystrophin function to skeletal muscles of mdx mice
21. Duchenne muscular dystrophy improved by Gentamycin
22. An agrin minigene rescues dystrophic symptoms in a mouse model for congenital muscular dystrophy