CenterPoints: Ana Maria Arbelaez, MD talks about her research
in children with type 1 diabetes within this Center.
Musculoskeletal and metabolic diseases are a leading cause of disability in children
and are frequently associated with genetic abnormalities that alter their normal
growth and development. Recent advances have led to the discoveries of some genetic
defects involved in several musculoskeletal and metabolic diseases, including those
that cause alterations in the skeleton (e.g. scoliosis, clubfoot), undergrowth syndromes
(e.g. celiac disease) and overgrowth syndromes (e.g. Beckwith Weidman disease).
The Center for Musculoskeletal and Metabolic Diseases will accelerate research through,
for example,the development of several strategies including international patient
registries and DNA banks for selected pediatric musculoskeletal and metabolic diseases
that will enable scientists to identify the
genes responsible for these diseases
and develop approaches to preventative measures within the next decade.
Additional work will be focused on the biology of tissue and organ growth, development
and regeneration that can lead to novel approaches to regenerative medicine and
progenitor cell therapy.