Scientists at the Icahn School of Medicine at Mount Sinai in New York, the National Center for Advancing Translational Sciences (NCATS), and elsewhere have reversed the effects of several life-threatening inherited neurodegenerative ...
Sep 8, 2022
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Adding to the growing body of literature demonstrating the feasibility of correcting lethal genetic diseases before birth, researchers at Children's Hospital of Philadelphia (CHOP) have used DNA base editing in a prenatal ...
Jul 13, 2021
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A team led by researchers at Baylor College of Medicine has uncovered an unexpected mechanism that can explain a form of Batten disease called neuronal ceroid lipofuscinosis 8. The findings published in the journal Nature ...
Nov 5, 2018
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Scientists propose in Nature blocking a molecule that drives inflammation and organ damage in Gaucher and maybe other lysosomal storage diseases as a possible treatment with fewer risks and lower costs than current therapies.
Feb 22, 2017
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Researchers at Baylor College of Medicine, Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital and King's College London have discovered a treatment that improves the neurological symptoms in a ...
Feb 6, 2017
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In a study of dogs, scientists showed that a new way to deliver replacement genes may be effective at slowing the development of childhood Batten disease, a rare and fatal neurological disorder. The key may be to inject viruses ...
Nov 11, 2015
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Researchers used blood platelets and bone marrow cells to deliver potentially curative gene therapy to mouse models of the human genetic disorder Hurler syndrome – an often fatal condition that causes organ damage and other ...
Feb 4, 2014
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Researchers have overcome a major challenge to treating brain diseases by engineering an experimental molecular therapy that crosses the blood-brain barrier to reverse neurological lysosomal storage disease in mice.
Feb 4, 2013
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Alzheimer's disease (AD) currently has no cure and is predicted to affect over 100 million people worldwide by 2050. Ongoing research is focused on two key neurotoxic proteins: amyloid beta (Aβ) and tau. While these proteins ...
Oct 17, 2022
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A mutation in the CLCN6 gene is associated with a novel, particularly severe neurodegenerative disorder. Scientists from the Leibniz-Forschungsinstitut für Molekulare Pharmakologie (FMP) and the Max Delbrück Center für ...
Nov 19, 2020
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