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Research Digest
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| Mar. 02 |
Muscle-Building Gene Protects Against Duchenne MD in Mice
Barton ER et al. Muscle-specific expression of insulin-like growth factor I counters muscle decline in mdx mice. J Cell Biol. 2002 Apr 1;157(1):137-48. [PubMed abstract] |
| Mar. 02 |
Anti-Macrophage' Drugs Could Work for DMD Wehling M et al. A nitric oxide synthase transgene ameliorates muscular dystrophy in mdx mice. J Cell Biol. 2001 Oct 1;155(1):123-31. [PubMed abstract] [Quest] |
| Nov. 01 |
Muscles Overcome by 'Junk' in Duchenne and Becker MDs?
Kolodziejczyk S et al. Activation of JNK1 contributes to dystrophic muscle pathogenesis. Curr Biol. 2001 Aug 21;11(16):1278-82. [PubMed abstract] [Quest] |
| Nov. 01 |
Better DNA Test Finds Virtually All Duchenne, Becker MD Mutations
Mendell, J et al. Diagnosis of Duchenne dystrophy by enhanced detection of small mutations. Neurology. 2001 Aug 28;57(4):645-50. [PubMed abstract] [Quest] |
| Nov. 01 |
Researchers find clue to dystrophin's function in brain
Sugita S et al. A stoichiometric complex of neurexins and dystroglycan in brain. J Cell Biol. 2001 Jul 23;154(2):435-45. [PubMed abstract] [MDA Technical Summary] |
| Nov. 01 |
Saving the diaphragm from DMD
Liu F et al. Transfer of Full-Length DMD to the Diaphragm Muscle of DMD (mdx/mdx) Mice through Systemic Administration of Plasmid DNA. Mol Ther. 2001 Jul;4(1):45-51. [PubMed abstract] [MDA News] [Quest] |
| Nov. 01 |
Cardiac exams suggested for Duchenne and Becker MD carriers
Grain L et al. Cardiac abnormalities and skeletal muscle weakness in carriers of Duchenne and Becker muscular dystrophies and controls. Neuromuscul Disord. 2001 Mar;11(2):186-91. [PubMed abstract] [Quest] |
| Nov. 01 |
New gene therapy strategy found in DMD
Kapsa R et al. In vivo and in vitro correction of the mdx dystrophin gene nonsense mutation by short-fragment homologous replacement. Hum Gene Ther. 2001 Apr 10;12(6):629-42. [PubMed abstract] [Quest] |
| Nov. 01 |
Oxandrolone has no benefit in DMD
Fenichel GM et al. A randomized efficacy and safety trial of oxandrolone in the treatment of Duchenne dystrophy. Neurology. 2001 Apr 24;56(8):1075-9. [PubMed abstract] [Quest] |
| Nov. 01 |
Gentamicin studies yield confusing results in MDs
Wagner KR et al. Gentamicin treatment of Duchenne and Becker muscular dystrophy due to nonsense mutations. Ann Neurol. 2001 Jun;49(6):706-11. [PubMed abstract] [Quest] |
| July 01 |
Cells coaxed to skip over genetic error in DMD mice
Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse. Proc Natl Acad Sci U S A. 2001 Jan 2;98(1):42-7. [PubMed abstract] [Quest] |
| July 01 |
Protein turns average mouse into mighty mouse Musaro A et al. Localized Igf-1 transgene expression sustains hypertrophy and regeneration in senescent skeletal muscle. Nat Genet. 2001 Feb;27(2):195-200. [PubMed abstract] [Quest] |
| July 01 |
Boosting integrin could be treatment for dystrophies Burkin DJ et al. Enhanced expression of the alpha 7 beta 1 integrin reduces muscular dystrophy and restores viability in dystrophic mice. J Cell Biol. 2001 Mar 19;152(6):1207-18. [PubMed abstract] [Quest] |
| July 01 |
Miniaturized gene raises hopes for Duchenne, Becker Wang B et al. Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model. Proc Natl Acad Sci U S A. 2000 Dec 5;97(25):13714-9. [PubMed abstract] [Quest] |
| July 01 |
Duchenne muscles may be starved for oxygen Sander M et al. Functional muscle ischemia in neuronal nitric oxide synthase-deficient skeletal muscle of children with Duchenne muscular dystrophy. Proc Natl Acad Sci U S A. 2000 Dec 5;97(25):13818-23 [PubMed abstract] [Quest] |
| July 01 |
A genetic modifier of Duchenne/Becker muscular dystrophy Rando TA et al. (2000): Rescue of dystrophin expression in mdx mouse muscle by RNA/DNA oligonucleotides. Proceedings of the National Academy of Sciences, 97(10):5363-5368. [PubMed abstract] [MDA Technical Summary] |
| July 01 |
Boys with DMD show specific memory deficits Hinton VJ et al. Poor verbal working memory across intellectual level in boys with Duchenne dystrophy. Neurology. 2000 Jun 13;54(11):2127-32. [PubMed abstract] [Quest] |
| July 01 |
Boosting utrophin to compensate for dystrophin Corbi N et al. The artificial zinc finger coding gene 'Jazz' binds the utrophin promoter and activates transcription. Gene Therapy 2000 Jun;7(12):1076-83. [PubMed abstract] [MDA Technical Summary] |
| Nov. 00 |
New Method Designed to Fix a Person's Own Copy of Dystrophin Gene Rando TA et al. (2000): Rescue of dystrophin expression in mdx mouse muscle by RNA/DNA oligonucleotides. Proceedings of the National Academy of Sciences, 97(10):5363-5368. [PubMed abstract] [Quest] |
| Nov. 00 |
and Bartlett RJ et al. (2000): Related Article In vivo targeted repair of a point mutation in the canine dystrophin gene by a chimeric RNA/DNA oligonucleotide. Nature Biotechnology, 18(6):615-22. [PubMed abstract] [Quest] |
| Nov. 00 |
New Strategy for Putting the Dystrophin Gene in the Safer AAV Vector Sun L et al. (2000): Overcoming adeno-associated virus vector size limitation through viral DNA heterodimerization. Nature Medicine, 6(5):599-602. [No Abstract Available [Quest] |
| Nov. 00 |
Making Utrophin Stand-in for Dystrophin Burton EA et al. (2000): A second promoter provides an alternative target for therapeutic up-regulation of utrophin in Duchenne muscular dystrophy. Proceedings of the National Academy of Sciences, USA. 96(24):14025-30. [PubMed abstract] [Quest] |
| Dec. 99 |
Electrical Recordings From Retina May Help Predict General Location of Dystrophin Gene Mutation Pillers DM, et al. (1999): Duchenne/Becker muscular dystrophy: correlation of phenotype by electroretinography with sites of dystrophin mutations. Human Genetics, 105:2-9. [PubMed abstract] |
| Dec. 99 |
No Longterm Psychosocial Problems Found in Women Tested for Duchenne Carrier Status in Childhood Jarvinen O, et al. (1999): Carrier testing of children for two X-linked diseases in a family based setting: a retrospective long term psychosocial evaluation. Journal of Medical Genetics, 36(8):615-620. [PubMed abstract] |
| Dec. 99 |
Up-regulation of Utrophin May be Associated With Blood Clots, Suggest Researchers* Higuchi I, et al. (1999): Multiple episodes of thrombosis in a patient with Becker muscular dystrophy with marked expression of utrophin on the muscle cell membrane. Acta Neuropathology, 98(3):313-316. * Note to Readers: This data, from a single case, shows a correlation only, not cause and effect [PubMed abstract] |
| Dec. 99 |
Stem Cells Generate Healthy Muscle in Mice With Muscular Dystrophy Gussoni E, et al.(1999): Dystrophin expression in the mdx mouse restored by stem cell transplantation. Nature, 401:390-394. (MDA-funded) [PubMed abstract] [Press release] [Quest] |
| Sept. 99 |
"Exon-skipping" May One Day Convert Duchenne MD to Becker Wilton SD, et al. (1999): Specific removal of a nonsense mutation from the mdx dystrophin mRNA using antisense oligonucleotides. Neuromuscular Disorders, 9(5):330-338. (MDA funded) [PubMed abstract] [MDA Technical Summary] |
| Sept. 99 |
Further Evidence for High Degree of Structural Conservation Between Utrophin and Dystrophin Wilson, J et al. (1999): Up71 and Up140, two novel transcripts of utrophin that are homologues of short forms of dystrophin. Human Molecular Genetics, 8(7): 1217-1278. [PubMed abstract] |
| Sept. 99 |
Most Duchenne and Becker Carriers Have Some Cardiac Abnormalities Hoogerwaard EM, et al. (1999): Cardiac involvement in carriers of Duchenne and Becker muscular dystrophy. Neuromuscular Disorders, 9:347-351. [PubMed abstract] |
| Sept. 99 |
Evidence for Signaling Defects in Muscular Dystrophy Grady M. et al. (1999): Role for alpha-dystrobrevin in the pathogenesis of dystrophin-dependent muscular dystrophies. Nature Cell Biology, 1:215-220. [PubMed abstract] |
| Sept. 99 |
Antibiotic May Allow Defective Dystrophin Gene to Generate Correct Protein Barton-Davis ER, et al. (1999): Aminoglycoside antibiotics restore dystrophin function to skeletal muscles of mdx mice. Journal of Clinical Investigation, 104(4):375-381. (MDA-funded) [PubMed abstract] [Press release] [F.A.Q.] [Quest] |
| Aug. 99 |
Utrophin Gene Therapy Effective in Mice That Lack Dystrophin Gilbert, R et al. (1999): Adenovirus-mediated utrophin gene transfer mitigates the dystrophic phenotype of mdx mouse muscles. Human Gene Therapy, 10:1299-1310. (MDA funded) [PubMed abstract] |
| Aug. 99 |
Deletion Helps Define Region of Dystrophin Gene Important for Mental Function Chen, DH et al. (1999): A novel deletion of the dystrophin S-promoter region cosegregating with mental retardation. Neurology, 52(3):638-640. [PubMed abstract] |
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