While Sangamo’s sickle cell partnership with Sanofi ground to a halt earlier this year, the California biotech’s Fabry disease program, still in its early stages, has been quietly chugging along. In Tuesday’s update on the gene therapy trial, adding two more patients from its last interim readout in November, Sangamo reported that it has begun...
Tag: <span>Gene Therapy</span>
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New gene therapy shows promise for treating age related macular degeneration
by Trinity College Dublin Rescue of ARPE19 cells insulted with NaIO3. 5.0 × 104 ARPE19 cells were transduced with AAV2/8-ophNdi1 5-h post-seeding; MOI = 5.4 × 105 (K–O). Twenty-eight-hour post-transduction cells were insulted with 5-mM NaIO3 (F–O) and 24-h post-insult cells were fixed and stained with Phalloidin-iFluor 647 (F-actin, light blue), and CPN60 (mitochondrial marker, magenta) and 8-OHdG-Alexa Fluor 488 (oxidative stress marker, green) immunocytochemistries;...
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Gene therapy for completely color blind children partly restores cone function
by University College London Credit: CC0 Public Domain Gene therapy has partly restored the function of the retina’s cone receptors in two children who were born completely color blind, reports a new study led by University College London (UCL) researchers. The findings, published in Brain, provide hope that the treatment is effectively activating previously dormant communication...
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Gene Therapy Approach Shows Promise in Treating ALS
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease characterized by the progressive loss of motor neurons in the brain and spinal cord responsible for voluntary movements and muscle control. In a new study, published in the journal Theranostics, researchers at University of California San Diego School of Medicine report that a gene therapy approach, developed at UC...
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New viral vectors for targeted gene therapy of heart muscle cells
by Medizinische Hochschule Hannover Graphical abstract. Credit: Molecular Therapy (2022). DOI: 10.1016/j.ymthe.2022.07.003 Gene therapies aim to cure severe, barely treatable monogentic diseases caused by a defect in a single gene. Medical hopes are correspondingly high. Some gene therapies have already been approved in Europe—for example, for spinal muscular atrophy (SMA), a congenital neuromuscular disease with severe muscle...
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How to turn muscle into a protein factory for advanced gene therapy
by Daegan Miller, University of Massachusetts Amherst Lila Gierasch (center), professor of biochemistry and molecular biology at UMass Amherst. Credit: UMass Amherst In a major new development in the quest to develop better gene therapies with which to treat a host of diseases, researchers at the University of Massachusetts Amherst and UMass Chan Medical School recently...
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Mouse study shows gene therapy may correct creatine deficiency disorder
by University of California, Los Angeles Credit: CC0 Public Domain A new study in mice finds that a gene therapy developed by a UCLA researcher appears to correct a rare creatine deficiency disorder that commonly results in intellectual disabilities, problems with speech, involuntary movements and recurrent seizures. The treatment potentially could represent an improvement upon...
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Bioprinting for bone repair improved with gene therapy
by A’ndrea Elyse Messer, Pennsylvania State University Bioprinting during surgery of bone constructs used as a controlled gene co-delivery platform for the repair of skull defects. Credit: Dong Heo, Kyung Hee University; Ozbolat Lab, Penn State / Penn State Given enough time and energy, the body will heal, but when doctors or engineers intervene, the...
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Gene therapy helps heal deadly ‘butterfly children’ blistering condition
by Dennis Thompson An experimental cream-based gene therapy may soon become the first U.S. government-approved means for treating a rare and devastating skin disease that produces “butterfly children.” Patients with recessive dystrophic epidermolysis bullosa (EB) are called butterfly children “because their skin is as fragile as the wings of a butterfly,” explained lead researcher Dr....
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Gene therapy for hemophilia A shows promise in phase 3 clinical trial
by Bob Yirka, Medical Xpress Graphical abstract. Credit: New England Journal of Medicine (2022). DOI: 10.1056/NEJMoa2113708 An international team of researchers has found more evidence that a new gene therapy may help some people with hemophilia A. In their paper published in the New England Journal of Medicine, the group describes the characteristics of the clinical trial and...