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
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
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
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
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
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
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
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
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
From October 6-8, clinicians, therapists and scientists gathered in Warsaw, Poland for the 2016 A-T Clinical Research Conference to discuss ways to improve the care for individuals with A-T. Highlights from the meeting included: An update on the CATNAP Study, a large pediatric neuroimaging study being performed in the UK and co-funded by the A-T … Read More