Long noncoding RNAs (lncRNAs) have been implicated in many biological processes.
However, due to the unique nature of lncRNAs and the consequential difficulties associated with …

Spinocerebellar ataxia type 7 is a polyglutamine disorder caused by an expanded CAG
repeat mutation that results in neurodegeneration. Since no treatment exists for this chronic …

The NF-κB pathway has critical roles in cancer, immunity and inflammatory responses.
Understanding the mechanism(s) by which mutations in genes involved in the pathway cause …

At Gene Therapy, we are continually monitoring the landscape of research, noting those
technologies which advance the goal of clinical translation of the field. In one of the most …

Dominantly inherited polyglutamine disorders are chronic neurodegenerative diseases
therapeutically amenable to gene-specific silencing strategies. Several compelling nucleic acid-…

An emerging realization of infectious disease is that pathogens can cause a high incidence
of genetic instability within the host as a result of infection-induced DNA lesions. These often …

Polyglutamine (polyQ) disorders are inherited neurodegenerative conditions defined by a
common pathogenic CAG repeat expansion leading to a toxic gain-of-function of the mutant …

Pharmaceuticals are indispensable to healthcare as the burgeoning global population is
challenged by diseases. The African continent harbors unparalleled genetic diversity, yet …

… Janine Scholefield … Kengo A , Nabeemeeah F , Denti P , Sabet R , Okyere-Manu G , Abraham
P , Weisner L , Mosala M H , Tshabalala S , Scholefield J , Resendiz-Galvan J E , Martinson …

On June 16th, Gene Therapy’s editorial team convened over a video call from all corners of
the globe and spent significant time discussing racism in science: what is happening, what …