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Research

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Methylmalonic Acidemia (MMA) Gene Therapy - Charles Venditti and Randy Chandler
Dr. Charles Venditti and Dr. Randy Chandler discuss recent developments in gene therapy for methylmalonic acidemia (MMA) - a group of inherited disorders in which the body is unable to process certai...
































Drs. Irini Manoli and Charles Venditti, Alyssa and Jennifer Sloan, Genetic Counselor~ during a June 2015 clinic visit at NIH







NIH News article on Gene Therapy and Methylmalonic Acidemia
    dated January 20, 2014:



A research team from the National Institutes of Health (NIH), led by Charles Venditti, M.D., Ph. D. Genetics, is conducting extensive research on mut 0 MMA.  Patients, predominantly children, from all over the world participate in the research studies.









Julien Sénac and Dr. Venditti in the research lab, 2011
 
 
Visit www.genome.gov/27529399 for more information about the clinical studies.
 
 
 
The Clinical Center at the NIH in Bethesda, Maryland is the nation's largest hospital devoted entirely to clinical research.  It is a national resource that makes it possible to rapidly translate scientific observations and laboratory discoveries into new approaches for diagnosing, treating, and preventing disease.
 
 
 
 
 
 
Dr. Charles Venditti, Bonnie, Alyssa and Denny Meisel, Dr. Irini Manoli and Jennifer Sloan, Genetic Counselor~ during a July 2013 clinic visit at NIH.  This photo was taken for a press release for a major breakthrough by the research team on kidney disease.
 
Find article entitled NIH Researchers Identify Therapy that May Curb Kidney Deterioration in Patients with Rare Disorder at http://www.genome.gov/27554473
 
 
At NIH, Dr. Venditti and his team have developed models of mice and other primitive organisms affected with MMA for manipulation in the laboratory.
 
 
The primary goal is to develop improved treatments and ultimately a cure for this metabolic disorder through gene, stem cell and small molecule drug therapies. 
 
For a list of published work related to the studies, visit www.genome.gov/19016902


 
 
Jennifer Sloan PhD, MS, Alyssa (Mut 0 MMA), Charles Venditti, M.D., Ph. D. Genetics and Kayleigh (Mut 0 MMA) during a 2011 visit to NIH for the clinical studies.
 

 

Angels for Alyssa entered into a donor agreement directly with the National Genome Research Institute on January 1, 2010 and has been responsible for the administration of the MMA Research Fund since that time.  Previously, the Organic Acidemia Association administered the MMA Research Fund.
 
 
Visit www.mmaresearch.com to learn more about the research efforts at NIH.
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Alyssa with
Dr. Venditti,
Jennifer Sloan PhD, MS
and her friend, Stephanie,
who also suffers
from MMA.
 
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