Therapeutic Antisense Targeting of Huntingtin | Zendy

Anne Smith | Zendy; Sarah J. Tabrizi | Zendy

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Therapeutic Antisense Targeting of Huntingtin

Author(s) -

Anne Smith,

Sarah J. Tabrizi

Publication year - 2019

Publication title -

dna and cell biology

Language(s) - Uncategorized

Resource type - Journals

SCImago Journal Rank - 0.895

H-Index - 77

eISSN - 1557-7430

pISSN - 1044-5498

DOI - 10.1089/dna.2019.5188

Subject(s) - huntingtin , biology , oligonucleotide , rna , huntingtin protein , huntington's disease , dna , disease , cancer research , computational biology , microbiology and biotechnology , genetics , gene , mutant , medicine , pathology

Antisense oligonucleotides (ASOs) are a relatively new therapeutic entity that utilizes short chemically modified strands of DNA in targeted interactions with RNA to modulate the type or amount of resultant protein. This brief review summarizes the preclinical, translational, and early clinical development of an ASO designed to reduce the production of the disease-causing protein in Huntington's disease, an inherited neurodegenerative disease.

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