Scientists are working to develop gene therapy to treat Batten disease, also known as Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL). This fatal, autosomal recessive neurodegenerative disorder results from a mutation in the gene that codes for the enzyme TPP1 (Tripeptidyl peptidase 1). In the absence of this enzyme, a substance called ceroid lipofuscin accumulates in lysosomes in the brain, resulting in seizures, blindness, decline in cognitive function and motor skills, dementia, and death by the late teens or early 20’s. The TPP1 gene is 6695 bp in length. Think about the characteristics of Batten disease, and then suggest an approach to gene therapy that might be effective for this specific genetic disorder. You may assume that your research team is working in the U.S. and your research is funded by a grant from the National Institutes of Health (NIH). 1. Briefly outline a procedure that you could use to carry out the gene therapy for Batten disease.
Scientists are working to develop gene therapy to treat Batten disease, also known as Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL). This fatal, autosomal recessive neurodegenerative disorder results from a mutation in the gene that codes for the enzyme TPP1 (Tripeptidyl peptidase 1). In the absence of this enzyme, a substance called ceroid lipofuscin accumulates in lysosomes in the brain, resulting in seizures, blindness, decline in cognitive function and motor skills, dementia, and death by the late teens or early 20’s. The TPP1 gene is 6695 bp in length. Think about the characteristics of Batten disease, and then suggest an approach to gene therapy that might be effective for this specific genetic disorder. You may assume that your research team is working in the U.S. and your research is funded by a grant from the National Institutes of Health (NIH).
1. Briefly outline a procedure that you could use to carry out the gene therapy for Batten disease.
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