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Tay-Sachs, Canavan, GM1 and Sandhoff diseases

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Latest Tay Sachs Disease News and Research

Study underscores the need for more precise rare disease definitions
Some diseases are like black swans. They occur so rarely that many physicians never encounter them in their clinical practice, complicating efforts to treat them.
New CRISPR genome “prime editing” system
Gene editing to correct genetic faults and removing the risk of hereditary diseases may soon become reality. Researchers have come up with a new gene editing technology that could help patients with sickle cell anaemia and up 89 percent of other genetic defects.
$28 million grant supports Cincinnati Children's role as coordinating center for Rare Diseases Clinical Research Network
The federal agency that oversees a large network of research centers seeking cures for hundreds of rare, disabling and often fatal diseases has named Cincinnati Children's as its new data management and coordinating center.

Latest Sandhoff Disease News and Research

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.
Researchers describe important step toward gene therapy for patients with Sandhoff disease
Babies with the rare, deadly genetic disorder Sandhoff disease begin to miss developmental milestones just months after birth. Lacking muscle tone, they never learn to sit up, develop heads too large to lift and eventually suffer uncontrollable seizures. There is no cure.
Genetic therapy for two inherited neurologic diseases moves closer to human clinical trials
Researchers at University of Massachusetts Medical School and Auburn University College of Veterinary Medicine are nearing human clinical trials on a genetic therapy for two rare neurological diseases that are fatal to children.

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

Gastruloids Stand In for Early Human Embryos

Remember human embryonic stem (hES) cells? We don’t hear much about them anymore. And so I was surprised to see an application…

The post Gastruloids Stand In for Early Human Embryos appeared first on DNA Science.
Two Views of Leeches, A Century Apart

Two genome sequences of the European medicinal leech Hirudo medicinalis have just been revealed in a pair of papers, and the unexpected…

The post Two Views of Leeches, A Century Apart appeared first on DNA Science.
How an Antibody Cocktail Against COVID-19 Channels the 3 Stooges – But is a Great Idea

“You imbecile!” bellowed Moe Howard as he stuck a finger up the nose of Curly. Moe the bully would often flick his…

The post How an Antibody Cocktail Against COVID-19 Channels the 3 Stooges – But is a Great Idea appeared first on DNA Science.

Latest Gangliosidosis News and Research

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.
New gene therapy shows promising results for treating neurodegenerative disorders
A new gene therapy approach designed to replace the enzyme that is deficient in patients with the inherited neurodegenerative disorders Tay-Sachs and Sandhoff diseases successfully delivered the therapeutic gene to the brains of treated mice, restored enzyme function, and extended survival by about 2.5-fold.
Researchers devise new genetic technique for IVF embryos
Researchers at the Johns Hopkins University School of Medicine have devised a new technique, which helps couples that are affected by or are carriers of genetic diseases have in vitro fertilized babies free of both the disease in question and other chromosomal abnormalities. The results were reported in the April issue of Fertility and Sterility.