University of Massachusetts

Edith L Pfister

Neil Aronin

Christian Mueller

Anastasia Khvorova

Julia Alterman

Ken Yamada

Faith Conroy

Phillip D Zamore

Novel oligonucleotides that enhance silencing of the expression of a gene containing a single nucleotide polymorphism (SNP) relative to the expression of the corresponding wild-type gene are provided. Methods of using novel oligonucleotides that enhance silencing of the expression of a gene containing a SNP relative to the expression of the corresponding wild-type gene are provided.

Modified oligonucleotides targeting SNPs

Aspects of the disclosure relate to compositions and methods useful for treating Huntington's disease. In some embodiments, the disclosure provides interfering nucleic acids (e.g., artificial miRNAs) targeting the huntingtin gene (HTT) and methods of treating Huntington's disease using the same.

AAV treatment of Huntington's disease

The present invention relates to the discovery of (SNPs) significantly associated with Huntington's disease (HD). The present invention utilizes RNA silencing technology (e.g. RNAi) against such SNPs optimally combined with select additional SNP targeting silencing agents, thereby resulting in an effective treatment of significantly-sized patient populations. Silencing agents having enhanced discriminatory properties are also featured.

Single nucleotide polymorphism (SNP) targeting therapies for the treatment of huntington'S disease

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Boxborough, MA, United States of America

Edith L Pfister