Myoblast implantation in Duchenne muscular dystrophy: The San Francisco study

Muscle and Nerve

Volumn 20, Issue 4, 1997, Pages 469-478

  Miller, R G ^a,b   Sharma, K R ^a   Pavlath, G K ^b   Gussoni, E ^b   Mynhier, M ^a   Yu, P ^a   Lanctot, A M ^b   Greco, C M ^a   Steinman, L ^b   Blau, H M ^b  

^a CALIFORNIA PACIFIC MEDICAL CENTER   (United States)

^b STANFORD UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

cell transplantation; cyclosporine; Duchenne muscular dystrophy; gene therapy; myoblasts

Indexed keywords

DYSTROPHIN; MESSENGER RNA;

ARTICLE; CELL TRANSPLANTATION; CHILD; CLINICAL ARTICLE; CLINICAL TRIAL; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; DUCHENNE MUSCULAR DYSTROPHY; HUMAN; IMPLANTATION; MALE; MUSCLE FORCE; MYOBLAST; POLYMERASE CHAIN REACTION; PRIORITY JOURNAL; TIBIALIS ANTERIOR MUSCLE;

ADULT; BIOPSY; CELL TRANSPLANTATION; CELLS, CULTURED; CHILD; CHILD, PRESCHOOL; CYCLOSPORINE; DYSTROPHIN; HISTOCOMPATIBILITY TESTING; HUMANS; IMMUNOSUPPRESSIVE AGENTS; MAGNETIC RESONANCE IMAGING; MALE; MUSCLE CONTRACTION; MUSCLE, SKELETAL; MUSCULAR DYSTROPHIES; PHYSICAL EXAMINATION; PROTEIN BIOSYNTHESIS; TISSUE DONORS; TRANSCRIPTION, GENETIC;

EID: 8244228688     PISSN: 0148639X     EISSN: None     Source Type: Journal    
DOI: 10.1002/(SICI)1097-4598(199704)20:4<469::AID-MUS10>3.0.CO;2-U     Document Type: Article

References (17)

1. Beauchamp JR, Morgan JE, Pagel CM, Partridge TA: Quantitative studies of the efficacy of myoblast transplantation. Muscle Nerve 1994;18(suppl 1):S261.
2. Blau HM, Dhawan J, Pavlath GK: Myoblasts in pattern formation and gene therapy. Trends Genet 1993;9:269-274.
3. Blau HM, Webster C: Isolation and characterization of human muscle cells. Proc Natl Acad Sci USA 1981;78:5623-5627.
4. Brooke MH, Fenichel G, Province MA, Griggs RC, Mendell JR, Moxley R, Miller JP, and the CIDD Group: Clinical investigation in Duchenne dystrophy: 2. Determination of the "power" of therapeutic trials based on the natural history. Muscle Nerve 1983;6:91-103.
5. Brooke M, Griggs R, Mendell J, Fenichel GM, Shumate JB, Pellegrino RJ: Clinical trial in Duchenne dystrophy: I. The design of the protocol. Muscle Nerve 1981;4:186-197.
6. Chamberlain JS, Gibbs RA, Rainier JE, Caskey CT: PCR protocols: a guide to methods and applications, in Innis MA, Gelfand DH, Sninsky JJ, White TJ (eds). San Diego, Academic Press, 1990, pp 272-281.
7. Cox GA, Cole NM, Matsumura K, Phelps SF, Hauschka SD, Campbell KP, Faulkner JA, Chamberlain JS: Overexpression of dystrophin in transgenic mdx mice eliminates dystrophic symptoms without toxicity. Nat Lett 1993;364:725-729.
8. Ellis JM, Nguyen thi Man: Specificity of dystrophin analysis improved with monoclonal antibodies [letter]. Lancet 1990;336:881-882.
9. Fradin MS, Ellis CM, Voorhees JJ: Management of patients and side effects during cyclosporine therapy for cutaneous disorders. J Am Acad Dermatol 1990;23:1265-1275.
10. 2 production in rat skeletal muscle in fever is signaled by a macrophage product distinct from interleukin 1 or other known monokines. J Clin Invest 1988;81:1378-1383.
11. Griggs RC, Moxley RT, Mendell JR, Fenichel GM, Brooke MH, Pestronk A, Miller JP, Cwik VA, Pandya S, Robison J, King W, Signore L, Schierbecker J, Florence J, Matheson-Burden N, Wilson B: Duchenne dystrophy: randomized, controlled trial of prednisone (18 months) and azathioprine (12 months). Neurology 1993;43:520-527.
12. Gussoni E, Pavlath GK, Miller RG, Lanctot AM, Sharma KR, Steinman L, Blau HM: Normal dystrophin transcripts detected in Duchenne muscular dystrophy patients after myoblast transplantation. Nature 1992;356:435-438.
13. Hardiman O, Sklar RM, Brown RH: Direct effects of cyclosporin A and cyclophosphamide on differentiation of normal human myoblasts in culture. Neurology 1993;43:1432-1434.
14. Hardiman O, Sklar RM, Brown RH: Methylprednisolone selectively affects dystrophin expression in human muscle cultures. Neurology 1993;43:342-345.
15. Huffman EP, Arahata K, Minetti C, Bonilla E, Rowland LP: Dystrophinopathy in isolated cases of myopathy in females. Neurology 1992;42:967-975.
16. Hoffman EP, Brown RH Jr, Kunkel LM: Dystrophin: the protein product of the Duchenne muscular dystrophy locus. Cell 1987;51:919-928.
17. Hoffman EP, Beggs AH, Koenig M, Kunkel LM, Angelini C: Cross-reactive protein in Duchenne muscle. Lancet 1989;ii: 1211-1212.