The General Hospital Corporation

Barry Kesner

Jeannie T Lee

Johnny Kung

Stefan Pinter

Ruslan Sadreyev

Michael Rosenberg

This invention relates to methods and compositions for selectively reactivating or downregulating certain genes, e.g., genes regulated by zinc-finger protein CCCTC-binding factor (CTCF) on autosomes (e.g., imprinted genes, tumor suppressors, cancer) and the inactive X chromosome (Xi), e.g., genes associated with X-linked diseases, e.g., Rett Syndrome, Factor VIII or IX deficiency, Fragile X Syndrome, Duchenne muscular dystrophy, and PNH, in heterozygous females carrying a mutated allele, in addition to a functional wildtype or hypomorphic allele.

CCCTC-binding factor (CTCF) RNA interactome

This invention relates to polycomb-associated RNAs, libraries and fragments of those RNAs, inhibitory nucleic acids and methods and compositions for targeting RNAs, and methods of use thereof.

RNA interactome of polycomb repressive complex 1 (PRC1)

Methods and compositions for selectively reactivating genes on the inactive X chromosome (Xi) in a locus-specific manner, e.g., genes associated with X-linked diseases, e.g., Rett Syndrome, Factor VIII or IX deficiency, Fragile X Syndrome, Duchenne muscular dystrophy, and PNH, in heterozygous females.

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Charlestown, MA, United States of America

Barry Kesner