Identifying Factors that Impact Growth in A-T

In collaboration with Valerie Natale, PhD of the Forgotten Diseases Research Foundation, the A-T Clinical Center at Johns Hopkins, funded by the A-T Children’s Project, is analyzing growth data for patients with A-T who have been followed over time to develop growth curves for A-T. The collaborators will then examine the effects of disease severity, … Read More

Sequencing RNA in Search of A-T Biomarkers

With support from the A-T Children’s Project, the lab of Sharon McGrath-Morrow, MD, a pediatric pulmonologist for the A-T Clinical Center, is sequencing RNA from A-T patient blood cells in hopes of identifying markers that will correlate with either immune problems or cancer and predict the disease’s course. If found and validated, such biomarkers could … Read More

Researchers Have Begun Accessing the Global A-T Family Data Platform

I’m pleased to report that we have already amassed health and genomic data on 265 children and young adults with A-T through our on-line Global A-T Family Data Platform. Contributing families span 25 countries, facilitated by the portal’s functionality in 10 languages. Now, research scientists have begun plunging into these de-identified data sets, seeking discoveries … Read More

YOU HELPED US REACH OUR MATCH GOAL!

What an incredible response we had to the Bill and Lydia Addy “Double Match” campaign! We are pleased to report that, with your help, we quickly received donations to cover the entire $125,000 that Bill and Lydia Addy had offered to match 2-to-1 on donations received by May 9th. As a result, combined with their … Read More

IMAGING THE CHANGING CEREBELLUM IN A-T

The A-T Children’s Project has recently awarded funding to Rob Dineen, PhD from the University of Nottingham in the UK to assemble existing brain imaging data and examine how the cerebellum changes in A-T. Results from the review may bring more clarity about where and when the most critical problems occur in the A-T brain, … Read More

TIMELINE AND PROGRESS FOR ASO GENE THERAPY

Email from Volunteer Chairman and Founder, Brad Margus: We’ve been overwhelmed by the generosity with which so many of you have reached out to support our efforts to treat, for the first time in history, a child with ataxia-telangiectasia (A-T) using antisense oligonucleotide gene therapy. This approach could slow or completely stop the disease in … Read More

Join the A-T CureTeam in Vegas or Florida

We know what you’re thinking …. You can hardly wait to sign up for your next A-T CureTeam marathon weekend! Well, you’re in luck. The marathon weekend registrations are up and running, just waiting for you to join in on the fun! Join A-T families and friends and raise funds this year for the first … Read More

ASO GENE THERAPY

Email from Volunteer Chairman and Founder, Brad Margus: In the 24 years that I’ve been seeking support for our battle to treat A-T, I’ve always erred on the side of caution in describing what we hoped to achieve… maybe a slight improvement in a day-to day task like swallowing, or a small enhancement to some … Read More

MAKING A-T STEM CELLS BROADLY AVAILABLE

  The A-T Children’s Project is making sure valuable cell models of A-T will be available to researchers everywhere. The breakthrough discovery in recent years that a patient’s cells could be reprogrammed into pluripotent stem cells — which have the capacity to produce many different cell types — has presented an enormous opportunity for A-T researchers. … Read More

REDUCING A-T SEVERITY BY TARGETING OTHER GENES

The laboratory of Stephen Jackson, PhD at the University of Cambridge in England has used CRISPR/Cas gene editing technology in cells to find genes that, when altered, compensate for the lack of the ATM protein. Now, with new funding from the A-T Children’s Project as well as Action for A-T, a UK-based non-profit organization, Dr. … Read More