People with the condition lack the protein dystrophin needed to keep muscle cells intact.
That can lead to life-threatening damage to the heart, and over time, death, often at a young age. The study tested two different dosages of the gene therapy in boys ranging in age from 6 to years old.
PPMD’s Gene Therapy Initiative
The higher tested dose appeared to be more effective, according to data presented at the Parent Project Muscular Dystrophy conference in Orlando, Florida. Pfizer said future trials will use the higher dose, which had triple the concentration of virus with genetic material. The U.
One had developed a kidney injury. So far, there are just two approved gene therapies in the United States, even though pharmaceutical companies have been pouring money into potentially lucrative treatments that could offer patients with deadly or debilitating conditions a possible one-time cure.
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It is known that the modular structure of dystrophin tolerates large internal deletions. This observation led to the development of two main therapeutic strategies: classical gene therapy with transfer of functional mini- or micro-dystrophin cDNAs in muscles, and targeted exon skipping. Exon skipping strategy, using antisense molecules or gene therapy with AAV-U7, converts an out-of-frame mutation into an in-frame mutation leading to an internally deleted dystrophin.
However, in preclinical DMD models, dystrophin restoration by AAV-U7-mediated exon-skipping therapy was shown to drastically decrease after one year in treated animals. We recently showed that pre-treating dystrophic mice muscle with a single dose of peptide-phosphorodiamidate morpholino PPMO antisense oligonucleotides led to transitory dystrophin expression at the sarcolemma and allowed efficient maintenance of AAV genomes enhancing significantly the long-term effect of AAV-U7 therapy.
These mice suffer from a much more severe and progressive muscle wasting, heart and diaphragm functions, impaired mobility and premature death, mimicking pathophysiology of DMD patients. Interestingly, emerging regulatory actors as lncRNA are localized in introns 44 and