Hey Everyone,
Pepsi is holding a project called Pepsi Refresh Project. Many great causes are being voted on each day to receive money for further research for disease, equipment needed, homeless and so on. One of the causes that I am asking YOU to vote for is personal to me because my 7 year old cousin suffers from this - Sanfilippo Syndrome. Below is a quick overview of this awful disease:
Sanfilippo syndrome is a genetic error of metabolism that affects children. It is a mucopolysaccharidoses, an MPS disorder, MPS-lll. Mucopolysaccharides are long chains of sugar molecules, used in building connective tissues. When the body is finished using these molecules, it breaks them down, with enzymes, and disposes of them. Children with MPS are missing or are deficient in the enzyme to break down the molecules. Instead, the body stores these molecules and this causes progressive damage.Thus, we plan to construct a vector plasmid for a self-complementary rAAV vector that creates the enzyme to break down the molecules. Once the vector is constructed, we will infect human MPS III skin fibroblasts cell cultures to determine the levels of expression and secretion of recombinant enzyme. After successfully replicating the vector, we will provide the GMP vector for preclinical studies and make this treatment available for all children impacted by this devastating disorder.
Pepsi is holding a project called Pepsi Refresh Project. Many great causes are being voted on each day to receive money for further research for disease, equipment needed, homeless and so on. One of the causes that I am asking YOU to vote for is personal to me because my 7 year old cousin suffers from this - Sanfilippo Syndrome. Below is a quick overview of this awful disease:
Sanfilippo syndrome is a genetic error of metabolism that affects children. It is a mucopolysaccharidoses, an MPS disorder, MPS-lll. Mucopolysaccharides are long chains of sugar molecules, used in building connective tissues. When the body is finished using these molecules, it breaks them down, with enzymes, and disposes of them. Children with MPS are missing or are deficient in the enzyme to break down the molecules. Instead, the body stores these molecules and this causes progressive damage.Thus, we plan to construct a vector plasmid for a self-complementary rAAV vector that creates the enzyme to break down the molecules. Once the vector is constructed, we will infect human MPS III skin fibroblasts cell cultures to determine the levels of expression and secretion of recombinant enzyme. After successfully replicating the vector, we will provide the GMP vector for preclinical studies and make this treatment available for all children impacted by this devastating disorder.
You can help this much needed money go to Sanfilippo Syndrom by voting each day via email, text, and through facebook. There is no cure and these children die young. Please, Please vote and help us get this much needed money so we can learn MORE about this disease and find a cure. VOTE NOW
