The SCID, Angels for Life Foundation, a patient advocacy group of the Primary Immune Deficiency Treatment Consortium (PIDTC), has announced the winners of its 2022 Grant Awards.
Donald Kohn, MD, of the University of California Los Angeles is awarded a one-year grant in the New and Innovative Curative Treatment Approach area of interest. Dr. Kohn’s proposal is titled “IL7Rα gene editing to treat IL7Rα-SCID.”
“The work being performed is to develop a gene editing approach to treat IL7Rα SCID starting with the initial studies to assess elements of the gene editing reagents,” the proposal states. “These studies will provide the basis for the next set of steps, to test the optimal IL7Rα gene editing reagents. The goal is to develop an effective method to treat IL7Rα SCID using gene editing in autologous HSC.”
Jolan Walter, MD, PhD, of the University of South Florida is awarded a one-year grant in the Innovative Approach Targeting a Marginalized Community area of interest. Dr. Walter’s proposal is titled “Predicting the clinical phenotype and increasing awareness of a novel RAG1 p.C176F founder variant causing atypical SCID with variable immune dysregulation in U.S. Mennonite communities.”
“The study involves members of conservative Mennonite communities with the same homozygous RAG1 p.C176F founder variant yet highly variable disease severity,” the proposal states. “These communities reside in four states: Pennsylvania, Virginia, North Carolina and Ohio. Conservative members of the Mennonite religion are marginalized regarding their health and healthcare needs for multiple reasons. The outcomes of the study are increased awareness of the RAG1 founder variant among Mennonite community members and greater inclination to seek genetic screening and follow-up care. Additionally, it is anticipated that the study will lead to novel preventive and therapeutic strategies that reduce disease severity of those who inherit the RAG1 founder variant as well as other variants of atypical SCID with variable clinical presentations.”
Learn more about the SCID, Angels for Life Foundation 2022 Grant Awards.
The Primary Immune Deficiency Treatment Consortium (PIDTC) is part of the Rare Diseases Clinical Research Network (RDCRN), which is funded by the National Institutes of Health (NIH) and led by the National Center for Advancing Translational Sciences (NCATS) through its Division of Rare Diseases Research Innovation (DRDRI). PIDTC is funded under grant number U54AI082973 as a collaboration between NCATS and the National Institute of Allergy and Infectious Diseases (NIAID).
