gene

Gene therapy research highlights X-linked RP associated with RPGR

Gene therapy research highlights X-linked RP associated with RPGR

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Reviewed by Michel Michaelides, MD Botaretigene sparoparvovec gene therapy (formerly AAV5-RPGR; MeiraGTx Holdings plc and Janssen Pharmaceuticals, Inc) for the treatment of a common and severe form of retinitis pigmentosa (RP), RPGR– associated X-linked RA, demonstrated improvements in retinal sensitivity and visual function compared to controls and had a manageable adverse event profile, according to …

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Prominent vet Lord Sandy Trees backs gene editing for healthier livestock

Prominent vet Lord Sandy Trees backs gene editing for healthier livestock

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This week’s Guest View is dedicated to veterinary professor Lord Trees who outlines his support for the Gene Technology (Precision Breeding) Bill – which is being heard in England – and its potential to deliver positive results for disease control, animal welfare and environmental impact. He points out, via Science for Sustainable Agriculture, that existing …

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Scientists have said having longer genes can make someone live longer (stock photo)

Scientists Unravel Mystery of Aging: Gene Length Is the Decisive Factor

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Scientists think they have cracked the secret of aging. Major genetic analysis of people, rodents and fish has revealed that the length of their DNA is directly related to their biological age. Shorter genes were associated with shorter lifespans, while longer genes were linked to better health and longevity. Scientists believe that if they can …

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Recently identified MECP2 gene mutation linked to congenital Rett syndrome...

Recently identified MECP2 gene mutation linked to congenital Rett syndrome…

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A new mutation has been discovered in the MECP2 gene – associated with most cases of Rett syndrome – in a toddler with clinical features suggestive of congenital variant-type Rett syndrome, one of the most severe atypical forms of the disease. The child had hypercapnia, or high levels of carbon dioxide in her blood, but …

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Putting STEM to Work: Efficient Gene Sequencing Through Automation

Putting STEM to Work: Efficient Gene Sequencing Through Automation

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Thanks to the generous support of Regeneron, River Journal publishes four times a year the on-the-spot report of a River Towns high school science student who is paired with a Regeneron scientist to find out what it’s like to put education STEM at work in the real world. Edmund Tsou is a junior at Briarcliff …

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The Y chromosome slowly disappears. A new sex gene could be the future of men

The Y chromosome slowly disappears. A new sex gene could be the future of men

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The sex of human babies and other mammals is determined by a male-determining gene on the Y chromosome. But the human Y chromosome degenerates and could die out in a few million years, leading to our extinction unless we develop a new sex gene. . The good news is that two rodent branches have already …

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Autism-linked gene discovered to shape nerve connections – Neuroscience News

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Summary: The autism-linked Gabrb3 gene appears to shape the formation of normal and atypical neural connections in the brain. Source: Cornell University A gene linked to autism spectrum disorders plays a critical role in early brain development and may shape the formation of normal and atypical nerve connections in the brain, according to a new …

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Promising study suggests AAV-based gene therapy can treat type I glutaric aciduria

Promising study suggests AAV-based gene therapy can treat type I glutaric aciduria

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Treatment of glutaric aciduria type I (GA-I) by intracerebroventricular administration of GCDH. Credit: Institute of Zoology, Chinese Academy of Sciences, China Glutaric aciduria type I (GA-I) is an inherited disease caused by a mutation in the glutaryl-CoA dehydrogenase (GCDH) gene. The global incidence of GA-I is estimated at 1/100,000, with approximately 75,000 cases worldwide. Patients …

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Promising study suggests AAV-based gene therapy can treat type I glutaric aciduria

Promising study suggests AAV-based gene therapy can treat type I glutaric aciduria

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Treatment of glutaric aciduria type I (GA-I) by intracerebroventricular administration of GCDH. Credit: Institute of Zoology, Chinese Academy of Sciences, China Glutaric aciduria type I (GA-I) is an inherited disease caused by a mutation in the glutaryl-CoA dehydrogenase (GCDH) gene. The global incidence of GA-I is estimated at 1/100,000, with approximately 75,000 cases worldwide. Patients …

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