Rett Syndrome: Crossing the Threshold to Clinical Translation.

ABSTRACT: Lying at the intersection between neurobiology and epigenetics, Rett syndrome (RTT) has garnered intense interest in recent years, not only from a broad range of academic scientists, but also from the pharmaceutical and biotechnology industries. In addition to the critical need for treatments for this devastating disorder, optimism for developing RTT treatments derives from a unique convergence of factors, including a known monogenic cause, reversibility of symptoms in preclinical models, a strong clinical research infrastructure highlighted by an NIH-funded natural history study and well-established clinics with significant patient populations. Here, we review recent advances in understanding the biology of RTT, particularly promising preclinical findings, lessons from past clinical trial

SUBMITTER: Katz DM 

PROVIDER: S-EPMC4924590 | biostudies-literature | 2016 Feb

REPOSITORIES: biostudies-literature