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Details: Last Updated: Monday, 18 November 2019 10:26

A collection of news from www.news-medical.net, an Internet resource.

News Feed: Tay-Sachs

Latest Tay Sachs Disease News and Research

New gene-editing method can correct many disease-causing mutations in mammalian cells
Some genetic disorders-such as cystic fibrosis, hemophilia and Tay Sachs disease-involve many mutations in a person's genome, often with enough variation that even two individuals who share the same disorder might have a different combination of mutations.
Dual vector gene therapy for GM2 gangliosidosis shows encouraging results in clinical trial
Patients in a Phase I/II clinical trial conducted by UMass Chan Medical School of a dual vector gene therapy for GM2 gangliosidosis, which includes Tay-Sachs and Sandhoff diseases, exhibited a biochemical correction of the disease with minimal adverse reactions.
Protein identified as key to preventing fat build-up and related diseases
Accumulation of fat molecules is detrimental to the cell. Researchers from the Yong Loo Lin School of Medicine, National University of Singapore (NUS Medicine), have made a breakthrough in understanding how our cells manage to stay healthy by recycling important fat molecules.

News Feed: Sandhoff

Latest Sandhoff Disease News and Research

Dual vector gene therapy for GM2 gangliosidosis shows encouraging results in clinical trial
Patients in a Phase I/II clinical trial conducted by UMass Chan Medical School of a dual vector gene therapy for GM2 gangliosidosis, which includes Tay-Sachs and Sandhoff diseases, exhibited a biochemical correction of the disease with minimal adverse reactions.
Targeted microglia replacement shows promise against fatal neurodegenerative conditions
Tay-Sachs and Sandhoff diseases, genetic disorders affecting the brain, have no effective treatment and are typically fatal within the first years of life.
Study identifies a potential treatment for Sandhoff and Tay-Sachs diseases
In a groundbreaking study, researchers at McMaster University have identified a potential treatment for Sandhoff and Tay-Sachs diseases—two rare, often fatal lysosomal storage disorders that cause progressive damage to nerve cells in the brain and spinal cord.

News Feed: Canavan Disease

Latest Canavan Disease (Leukodystrophies) News and Research

Human stem cells show promise against fatal children's diseases
Scientists have used human stem cells to dramatically improve the condition of mice with a neurological condition similar to a set of diseases in children that are invariably fatal, according to an article in the June issue of the journal Cell Stem Cell.

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News Feed: DNA Science

Geneticist Ricki Lewis blogs from the cutting edge of genomics, including genetic testing, stem cells, gene therapy and more.

Do Cats Get Dementia?

Babycat may have been a “naturally occurring model of Alzheimer’s disease,” according to a new report in The European Journal of Neuroscience…

The post Do Cats Get Dementia? appeared first on DNA Science.
FDA’s New Plausible Mechanism Guidance for Implementing Gene Editing and RNA Therapies is Good News for the Rare Disease Community

Last year, nine-month-old KJ Muldoon made history when a variation of CRISPR gene editing, called base editing, swapped one DNA building block…

The post FDA’s New Plausible Mechanism Guidance for Implementing Gene Editing and RNA Therapies is Good News for the Rare Disease Community appeared first on DNA Science.
Organoids Model Spinal Cord Injuries

Organoids are tiny bits of organs nurtured in lab glassware from stem cells. I joke about them at Halloween, when a few…

The post Organoids Model Spinal Cord Injuries appeared first on DNA Science.

News Feed: Gangliosidosis-1

Latest Gangliosidosis News and Research

Dual vector gene therapy for GM2 gangliosidosis shows encouraging results in clinical trial
Patients in a Phase I/II clinical trial conducted by UMass Chan Medical School of a dual vector gene therapy for GM2 gangliosidosis, which includes Tay-Sachs and Sandhoff diseases, exhibited a biochemical correction of the disease with minimal adverse reactions.
ChatGPT: A diagnostic sidekick for doctors? Caution advised for non-professionals
Research evaluates the diagnostic accuracy of ChatGPT, an AI language model, in providing medical advice online. It concludes that ChatGPT can be a useful tool for assisting human doctors in diagnosing difficult cases, but non-professionals should use it with caution.
Gene therapy is the best stratagem to impact the course of GM2 gangliosidosis
Since its clinical description in 1881, Tay-Sachs disease has had almost totemic significance as a cruel and relentless genetic condition that destroys the developing brain of babies and young children.

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A collection of news from www.news-medical.net, an Internet resource.

News Feed: Tay-Sachs

News Feed: Sandhoff

News Feed: Canavan Disease

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News Feed: DNA Science

News Feed: Gangliosidosis-1

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