Children's Medical Center Corporation

Harvard College

The General Hospital Corporation

The General Hospital Corporation D/b/a Massachusetts General Hospital

Wataru Ebina

Derrick J Rossi

Kenneth R Chien

Lee L Rubin

Morag Helen Stewart

Lior Zangi

Lance Davidow

Paula Gutierrez-Martinez

Leon M Ptaszek

Oi-Lan Lui

Kathy Oi-Lan Lui

Oi Lan Lui

Aspects of the invention described herein relate to synthetic, modified RNAs and their use in vivo to modulate gene expression. Aspects of the invention further relate to the use of these synthetic, modified RNAs in myocytes, cardiomyocytes, and tumors.

Efficient protein expression in vivo using modified RNA (mod-RNA)

The methods and compositions described herein relate to producing, expanding, enriching, and/or maintaining hematopoietic stem cells ex vivo by treating the cells with an agent(s) that exhibits two or more activities selected from modulation of histone methylation; inhibition of TGFβ signaling; inhibition of p38 signaling; activation of canonical Wnt signaling; and modulation of histone acetylation. In some embodiments, the technology described herein relates to transplantation of hematopoietic stem cells.

Methods and compositions relating to hematopoietic stem cell expansion, enrichment, and maintenance

The methods and compositions described herein relate to producing, expanding, enriching, and/or maintaining hematopoietic stem cells ex vivo by treating the cells with an agent(s) that exhibits two or more activities selected from modulation of histone methylation; inhibition of TGF&#x3b2; signaling; inhibition of p38 signaling; activation of canonical Wnt signaling; and modulation of histone acetylation. In some embodiments, the technology described herein relates to transplantation of hematopoietic stem cells.

Aspects of the invention described herein relate to synthetic, modified RNAs and their use in vivo to modulate gene expression. Aspects of the invention further relate to the use of these synthetic, modified RNAs in myocytes, cardiomyoctes, and tumors.

Efficient protein expression in vivo using modified RNA (MOD-RNA)

Growing community of inventors

Boston, MA, United States of America

Wataru Ebina