Question

What did you learn about how muscular dystrophy impacts muscle fiber and sarcomere physiology? What did you learn about how muscular dystrophy and gene therapy's impact on proteins produced by our cells?

Answer 1

Muscular dystrophy affects muscle fiber physiology by causing muscle weakness and degeneration due to a lack of dystrophin protein. Gene therapy aims to increase production of utrophin as a potential treatment for muscular dystrophy.

Step-by-step explanation:

In Duchenne muscular dystrophy (DMD), a lack of the protein dystrophin leads to muscle weakness and degeneration.

Dystrophin helps the thin filaments of myofibrils bind to the sarcolemma, the muscle cell membrane.

Without sufficient dystrophin, muscle contractions cause the sarcolemma to tear, which results in an influx of calcium ions and cellular damage.

Over time, this leads to muscle fiber degradation and functional impairments.

Gene therapy is being explored as a potential treatment for muscular dystrophy.

One approach involves boosting the production of utrophin, a protein similar to dystrophin that can assume its role in preventing cellular damage.

This may help compensate for the lack of dystrophin and improve muscle function