Category Biology/Biotechnology

New Therapy Halts progression of ALS/ Lou Gehrig’s disease in mice

Copper, zinc superoxide dismutase is essential to life, but when damaged can become toxic. Credit: Photo courtesy of Oregon State University

Copper, zinc superoxide dismutase is essential to life, but when damaged can become toxic. Credit: Photo courtesy of Oregon State University

OSU researchers have announced that they have essentially stopped the progression of amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease, for nearly 2 years in one type of mouse model used to study the disease – allowing the mice to approach their normal lifespan. In decades of work, no treatment can do anything but prolong human survival less than a month in ALS. This mouse model is one that may more closely resemble the human reaction to this treatment, using copper-ATSM. Researchers are moving as quickly as possible toward human clinical trials, testing first for safety and then efficacy.

ALS is known to be caused by the death and deterior...

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Discovered: How to Unlock Inaccessible Genes

This image shows the binding profile of chromatin remodelers on 12,000 distinct genes. The researchers used bioinformatics tools to visualize a great number of genes, here aligned by their promoters and arranged according to their nucleosome distribution. Remodelers attached to nucleosomes (dark blue verticals lines) are present on each side of the gene promoter. The researchers identified two distinct nucleosomal architectures (represented by drawings at top and bottom). Researchers demonstrated that remodeler activity depends on this nucleosome organization: some are more specifically required for expression of genes at nucleosome-dense promoters (upper part), whereas others act preferentially on promoters with a low nucleosomal density (bottom part). Credit: Matthieu Gérard, University of Paris-Sud

This image shows the binding profile of chromatin remodelers on 12,000 distinct genes. The researchers used bioinformatics tools to visualize a great number of genes, here aligned by their promoters and arranged according to their nucleosome distribution. Remodelers attached to nucleosomes (dark blue verticals lines) are present on each side of the gene promoter. The researchers identified two distinct nucleosomal architectures (represented by drawings at top and bottom). Researchers demonstrated that remodeler activity depends on this nucleosome organization: some are more specifically required for expression of genes at nucleosome-dense promoters (upper part), whereas others act preferentially on promoters with a low nucleosomal density (bottom part)...

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CRISPR used to Repair Blindness-causing Genetic Defect in Patient-derived Stem Cells

Correction of RPGR mutation in patient iPSC line.

Correction of RPGR mutation in patient iPSC line.

CUMC and Uni of Iowa scientists have used CRISPR, to repair a genetic mutation responsible for retinitis pigmentosa (RP), an inherited condition that causes the retina to degrade and leads to blindness in at least 1.5 million cases worldwide. It marks the first time researchers have replaced a defective gene associated with a sensory disease in stem cells that were derived from a patient’s tissue.

“Our vision is to develop a personalized approach to treating eye disease,” says A/Prof Stephen Tsang, MD, PhD. “We still have some way to go, but we believe that the first therapeutic use of CRISPR will be to treat an eye disease. Here we have demonstrated that the initial steps are feasible...

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Research hints at a Nutritional Strategy for Reducing Autism Risk

Highlights •TMLHE controls the neural stem cell (NSC) pool in the embryonic mouse neocortex •CPT1A and fatty acid mobilization from lipid droplets regulate the NSC pool •TMLHE deficiencies lead to increased symmetric differentiating division of NSCs •NSC defects under TMLHE deficiencies can be rescued by exogenous carnitine

Highlights •TMLHE controls the neural stem cell (NSC) pool in the embryonic mouse neocortex •CPT1A and fatty acid mobilization from lipid droplets regulate the NSC pool •TMLHE deficiencies lead to increased symmetric differentiating division of NSCs •NSC defects under TMLHE deficiencies can be rescued by exogenous carnitine

Besides folic acid, another prenatal supplement could protect against a certain type of autism, according to research: carnitine. It is required for transport of fatty acids into mitochondria – that converts these fats into energy...

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