Tag : inherited

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Scientists identify 'modifier gene' that determines severity of inherited kidney disease

Newsemia
Scientists have developed a new way to understand complex genetic diseases and have identified a gene that modifies the severity of inherited kidney disease, paving...
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New NIH program aims to improve lives for people with inherited blood and immune diseases

Newsemia
People with inherited diseases of the blood and immune system can now receive treatment at the National Institutes of Health Clinical Center through a new,...
Pediatrics

Rare inherited enzyme disorder yields insight into fibrosis

Newsemia
St. Jude investigators have discovered an association between a deficiency in the enzyme neuraminidase 1 and the build-up of connective tissue in organs such as...
Biology

IVERIC bio Enters into Strategic Manufacturing Relationship with Catalent’s Paragon Gene Therapy for Orphan Inherited Retinal Diseases

Newsemia
NEW YORK and SOMERSET, N.J. (PRWEB) June 17, 2019 IVERIC bio, Inc. (NASDAQ: ISEE) and Catalent Biologics, today announced that they have entered into an...
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Learned avoidance behavior can be inherited for multiple generations, say Princeton researchers

Newsemia
Princeton University researchers have discovered that learned behaviors can be inherited for multiple generations in C. elegans, transmitted from parent to progeny via eggs and...
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Scientists use gold nanoparticles to edit genes tied to HIV, inherited blood disorders

Newsemia
Scientists at Fred Hutchinson Cancer Research Center took a step toward making gene therapy more practical by simplifying the way gene-editing instructions are delivered to...
Pediatrics

Italian translation, cultural adaptation, and pilot testing of a questionnaire to assess family burden in inherited ichthyoses

Newsemia
Inherited ichthyoses are rare disorders characterized by generalized skin scaling. Among them, autosomal recessive congenital ichthyoses (ARCI) form a major subgroup presenting lifelong and severely...
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Unique gene therapy approach paves new way to tackle rare, inherited diseases

Newsemia
Nonsense mutations are single-letter errors in the genetic code that prematurely halt the production of critical proteins. These unfinished proteins are unable to function normally,...

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