University of Massachusetts

Other

Scriptgen Pharmaceuticals, Inc.

Fulcrum Therapeutics, Inc.

Michael R Green

Sanchita Bhatnagar

Guangping Gao

Manas Kumar Santra

Minggang Fang

Narendra Wajapeyee

Peter L Jones

Maria L Zapp

Jose G Teodoro

Miguel Sena Esteves

James W Lillie

Dan Wang

Seth Stern

Gerald V Denis

Richard K Boice

Destin Heilman

Joseph C Reese, Jr

Christian C Fritz

Susanne Wagner

Laxminarayana Devireddy

Walter Kowtoniuk

Fabian Richard

Tessa Mercedes Simone

Leyuan Ma

Giovanni Perini

Methods that include inhibition of ROCK1/2 and other protein kinases to treat Friedreich Ataxia.

Inhibition of protein kinases to treat Friedreich ataxia

The disclosure relates to methods and compositions for regulating expression of DUX4. In some aspects, methods described by the disclosure are useful for treating a disease associated with aberrant DUX4 expression (e.g., facioscapulohumeral muscular dystrophy).

Therapeutic targets for facioscapulohumeral muscular dystrophy

Methods of treating subjects having disorders associated with mutations in the FXN gene and/or having reduced expression of frataxin protein, e.g., Friedreich ataxia (FA). Generally, the methods include administering a therapeutically effective amount of an agent that increases expression of frataxin protein as described herein, e.g., an inhibitor of a FXN Repressing Factor (FXN-RF).

Genetic and pharmacological transcriptional upregulation of the repressed FXN gene as a therapeutic strategy for Friedreich ataxia

The disclosure relates to methods and compositions for reactivating a silenced FMR1 gene. In some aspects, methods described by the disclosure are useful for treating a FMR1-inactivation-associated disorder (e.g., fragile X syndrome).

Compositions and methods for modulating FMR1 expression

In some aspects, the disclosure relates to compositions and methods of engineering a transgene. In some embodiments, the disclosure provides self-regulating recombinant nucleic acids, viral vectors and pharmaceutical compositions comprising a MeCP2 transgene. In some embodiments, compositions and methods described by the disclosure are useful for treating diseases and disorders associated with a loss of function mutation, for example Rett syndrome.

Self-regulating AAV vectors for safe expression of MeCP2 in rett syndrome

Methods of treating a tumor in a subject include identifying a subject having, at risk for, or suspected of having a tumor, and administering to the subject an effective amount of an SRPX.

SRPX for treatment of cancer

In some aspects, the disclosure relates to the reactivation of inactive X chromosomes (Xi). In some embodiments, the disclosure provides compositions and methods for the reactivation of inactive X chromosomes. In some embodiments, the compositions and methods described by the disclosure may be useful for the treatment of dominant X-linked diseases.

Compositions and methods for Xi chromosome reactivation

Compositions and methods for XI chromosome reactivation

Methods for treating chronic myeloid leukemia (CML), e.g., BCR-ABL inhibitor imatinib mesylate (IM)-resistant CML, using combination treatments, e.g., combined treatment with a BCR-ABL inhibitor, e.g., IM, and a MEK inhibitor, e.g., trametinib.

Treating chronic myelogenous leukemia (CML)

A dolly for transporting goods. The dolly includes a frame, a wheel assembly mounted on the frame, and a baseplate extending from the frame. The baseplate has a horizontal portion extending forward from the frame and a vertical portion extending upward from the horizontal portion. The dolly includes a handgrip attached to the frame for maneuvering the dolly and an upper support bar removably attached to and extending laterally across the frame at a pre-determined height above the baseplate. The upper support bar having a length greater than the width of the frame. The dolly includes a lower support bar removably attached to and extending laterally across the vertical portion of the baseplate. The lower support bar has a length greater than the width of the baseplate.

System for increasing dolly capacity

Growing community of inventors

Boylston, MA, United States of America

Michael R Green