Molecular-targeted therapy for Duchenne muscular dystrophy: progress and potential
Scimè A, Rudnicki MA.
Regenerative Medicine Program,
Ottawa Health Research Institute,
Ottawa, Ontario, Canada.
Mol Diagn Ther. 2008;12(2):99-108.


Duchenne muscular dystrophy (DMD) is a lethal heritable childhood myodegenerative condition caused by a mutation within the gene encoding the dystrophin protein within the X chromosome. While, historically, patients with this condition rarely lived into their thirties, they are now living substantially longer as a result of new treatments based on multi-disciplinary care. Despite these advances, the prognosis for DMD patients is limited, and a progressive reduction in quality of life and early death in adulthood cannot be prevented using currently available treatment regimens. The best hopes for a cure lies with cellular and gene therapy approaches that target the underlying genetic defect. In the past several years, viral and nonviral gene therapy methodologies based on adeno-associated viruses, naked plasmid delivery, antisense oligonucleotides, and oligonucleotide-mediated gene editing have advanced to a high degree of sophistication, to the extent that research has moved from the laboratory setting to the clinic. Notwithstanding these accomplishments, shortcomings with each therapy remain, so more work is required to devise an appropriate therapeutic strategy for the management and eventual cure of this debilitating disease.
Muscular dystrophy
Genetic moral enhancement
Germline genetic engineering
Preimplantation genetic diagnosis
A life without pain? Hedonists take note'
'The Principle of Procreative Beneficience'
Do the DREAMless learn more and age less?
Gene therapy and performance enhancement
'Everybody in the world is my friend' hypersociability in Williams syndrome

and further reading

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Utopian Surgery?
The Good Drug Guide
The Abolitionist Project
The Hedonistic Imperative
The Reproductive Revolution
MDMA: Utopian Pharmacology
Critique of Huxley's Brave New World