A community dedicated to finding a cure for GNE Myopathy
Advances in Hereditary Inclusion Body Myopathy
GNE Myopathy (aka Hereditary Inclusion Body Myopathy or HIBM) is an ultra rare genetic disorder that affects roughly 1 in 5 million individuals worldwide.
Patients with GNE Myopathy suffer from progressive muscle weakness and unfortunately become wheelchair bound as the disease progresses. HIBM is highly prevalent in the Iranian Jewish community, with almost 1 in 10 individuals having a single copy of the mutated gene known as M712T (or M743T).
We are collaborating with some of the world’s leading experts in order to streamline and advance the most cutting edge discoveries from the laboratory to the clinic.
A decade ago if someone talked about inserting a gene into a human cell, or removing and correcting a single mistake in our DNA with surgical precision, most scientists would have laughed. What was science fiction then is reality now.
Join us on this journey and follow our progress as we continue to update the community with our latest developments and success stories.
Johns Hopkins University School of Medicine Researchers spearheaded by Dr. Greg Newby are the recipients of a multi-year grant from Solve GNE.
Using latest-generation CRISPR tools – base editors and prime editors – the team will attempt to directly correct the genetic mutation in laboratory models.
Advancing a new therapy through human clinical trials is a complex, expensive and time consuming process that involves the coordinated efforts of patients and their families, physicians, scientists and federal officials.
This mountain may seem tall and out of reach but it’s certainly not impossible to summit.
With careful planning and proper funding we CAN find a path forward and thanks to the latest advances in molecular biology, we can reach our goal in a reasonably short period of time.