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

APPLYING MACHINE LEARNING TO A-T

With an undergraduate degree in electrical engineering, medical training in neurology, and a PhD in robotics, Anoopum Gupta, MD, PhD at Massachusetts General Hospital is unusually qualified to apply new technologies to unravel complicated neurological problems like A-T. Supported by the A-T Children’s Project, Dr. Gupta has begun training a computer system to analyze video, … Read More

A-T FAMILIES PROVIDING DATA TO RESEARCHERS

A year has passed since the launch of the Global A-T Family Data Platform, an effort overseen by A-T families to enable researchers and clinicians to mine large data sets and gain new insights about ataxia-telangiectasia. So far, A-T families from 19 countries have contributed health information about their children who have A-T. The platform’s … Read More

Targeting Neutrophil-driven Inflammation to Reduce Lung Problems in A-T

The A-T Children’s Project has begun exploring whether anti-inflammatory drugs might prevent or reduce lung problems that can become life-threatening for people with ataxia-telangiectasia. The organization has awarded funding to JoAnn Sekiguchi, PhD at the University of Michigan to determine if anti-inflammatory compounds can reduce lung damage in a mouse model of A-T. About 25 … Read More

A-T IN LATIN AMERICA

The A-T Children’s Project is funding Beatriz Tavares Costa-Carvalho, MD and her colleagues to learn more about A-T in the Latin American population with a new grant entitled Ataxia-Telangiectasia: Epidemiological Survey in Latin America. Studying how a disease affects people over time is important not just to understanding the nature of the disease and its … Read More

INFLAMMATION, SWALLOWING, AND AGING IN A-T

Sharon McGrath-Morrow, a pediatric pulmonologist, published results showing that higher levels of IL-6 and IL-8 in the blood, markers for inflammation, are associated with poorer lung function in people with A-T, suggesting a link between inflammation and lung decline in this disease. Maureen Lefton-Greif, a speech and swallowing specialist, published results demonstrating abnormal coupling of … Read More