Multiplicity of experimental approaches to therapy for genetic muscle diseases and necessity for population screening.

Journal of Muscle Research and Cell Motility
Nigel G Laing

Abstract

Currently a multiplicity of experimental approaches to therapy for genetic muscle diseases is being investigated. These include replacement of the missing gene, manipulation of the gene message, repair of the mutation, upregulation of an alternative gene and pharmacological interventions targeting a number of systems. A number of these approaches are in current clinical trials. There is considerable anticipation that perhaps more than one of the approaches will finally prove of clinical benefit, but there are many voices of caution. No matter which approaches might ultimately prove effective, there is a consensus that for most benefit to the patients it will be necessary to start treatment as early as possible. A consensus is also developing that the only way to do this is to implement population-based newborn screening to identify affected children shortly after birth. Population-based newborn screening is currently practised in very few places in the world and it brings with it implications for prevention rather than cure of genetic muscle diseases.

References

May 20, 1978·Lancet·D Gardner-MedwinS Green
Jul 8, 1978·Lancet·R BeckmannG Scheuerbrandt
Jan 1, 1988·Archives of Neurology·A D Roses
Jan 1, 1993·Molecular and Cell Biology of Human Diseases Series·N G Laing
Feb 6, 1993·BMJ : British Medical Journal·D M BradleyA J Clarke
Jul 1, 1997·Neuromuscular Disorders : NMD·B A Kakulas
Dec 12, 2001·Neuromuscular Disorders : NMD·Victor Dubowitz
Mar 8, 2002·Archives of Disease in Childhood. Fetal and Neonatal Edition·E P ParsonsD M Bradley
Oct 2, 2002·Human Molecular Genetics·Melissa J Spencer, Ronald L Mellgren
Dec 24, 2002·Archives of Disease in Childhood·E P ParsonsA J Clarke
Jan 23, 2004·The American Journal of Bioethics : AJOB·John A Robertson
May 4, 2004·European Journal of Paediatric Neurology : EJPN : Official Journal of the European Paediatric Neurology Society·Evelyn P ParsonsDon M Bradley
Oct 29, 2004·Molecular Therapy : the Journal of the American Society of Gene Therapy·Thea ShavlakadzeMiranda D Grounds
Oct 26, 2005·Nature Biotechnology·Yi LaiDongsheng Duan
Apr 4, 2006·Journal of Human Genetics·Louis M KunkelLeta Steffen
Jul 29, 2006·Journal of Muscle Research and Cell Motility·Dominic J Wells
Aug 26, 2006·Midwifery·Evelyn P ParsonsDon M Bradley
Nov 17, 2006·Nature·Maurilio SampaolesiGiulio Cossu
Dec 15, 2006·Gene Therapy·H Herweijer, J A Wolff
Jan 17, 2007·Genome Biology·Elaine R Mardis
Feb 3, 2007·Proceedings of the National Academy of Sciences of the United States of America·Helge AmthorKetan Patel
Apr 24, 2007·Nature·Ellen M WelchH Lee Sweeney
Jul 21, 2007·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Christian S KuhrRainer Storb
Aug 2, 2007·Molecular Therapy : the Journal of the American Society of Gene Therapy·Jatinderpal R DeolGeorge Karpati
Oct 24, 2007·Neuromuscular Disorders : NMD·Steve Wilton
Dec 28, 2007·The New England Journal of Medicine·Judith C van DeutekomGert-Jan B van Ommen
Jan 22, 2008·Neuromuscular Disorders : NMD·Hannah G RadleyMiranda D Grounds
Jan 25, 2008·Clinical and Experimental Pharmacology & Physiology·Miranda D GroundsThea Shavlakadze
Feb 7, 2008·Human Molecular Genetics·Marc BartoliIsabelle Richard
Feb 7, 2008·The Cochrane Database of Systematic Reviews·A Y ManzurA Swan

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Citations

Jan 30, 2015·European Journal of Human Genetics : EJHG·Siaw H WongSylvia A Metcalfe
Jul 31, 2014·European Journal of Human Genetics : EJHG·Sally LawtonSylvia A Metcalfe
Apr 4, 2012·Critical Reviews in Clinical Laboratory Sciences·Nigel G Laing

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