Disease and Molecular Basis

Paper Summary:

This paper examines how there are two main categories of disease for which therapies are currently being investigated at the molecular level. These two categories of disease are genetic and infectious. It discusses cystic fibrosis and muscular dystrophy at the molecular level and looks at possible gene therapy treatments.

From the Paper:

"Therapies for muscular dystrophies are at the moment only for symptomatic treatment, but advances for curing them are being researched. A pharmacological therapy addresses the pathophysiological changes that lead to muscle cell death. Transplantation therapy involves the transfer of myoblasts from an unaffected donor into the muscle of an affected patient. The myoblasts fuse and become part of the patient's own muscle tissue. Gene therapy uses retroviral, adenoviral and herpes vectors to transfer the correct functional DMD gene into patients. One problem with retrovirus is that they are not big enough to carry the 11kb gene, so it has been found that you can cut out some of the coding region to reduce its size, but without affecting its function."

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