Overview
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.
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.
How will the 250K be Used? | |
| Budget Notes: The award money will NOT be used for any animal testing. | |
| $ 75,000 | Production, optimization & validating of vector |
| $ 75,000 | Perform studies on patient fibroblast cells |
| $ 100,000 | Production of clinical grade vector |
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