Tag: rare diseases

Medical illustration showing CRISPR gene editing process for hereditary angioedema treatment
New StudiesResearch Digest

First In Vivo CRISPR Gene Editing Treatment Shows Promise for Rare Blood Disorder

First clinical trial of in vivo CRISPR gene editing for hereditary angioedema shows promising results. The investigational therapy targets genetic…

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GMJ Research Desk
Medical illustration showing neural pathways and gene function in movement disorders
New StudiesResearch Digest

Hidden Immune Gene Linked to Rare Neurological Movement Disorder

Scientists discovered that CD99L2, a gene previously linked only to immune function, causes rare movement disorders. Analysis of nearly 3,000…

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GMJ Research Desk

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