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READING PART B,QUESTION NO 1

Mauscular Dystrophies.



Muscular dystrophies are monogenetic diseases that are often characterized by the degeneration of both cardiac and skeletal muscle. Gene therapy to correct the mutated gene has shown promise in both animal models and clinical trials; however, current gene delivery strategies are limited to the introduction of the corrected gene into only one tissue. Strategies to target multiple striated muscle types would provide a much-needed improvement for the treatment of muscular dystrophies. 

Muscular dystrophies are inherited genetic diseases that are particularly challenging from a therapeutic point of view. As striated muscles are formed by syncytial postmitotic cells that have limited or absent regenerative potential, muscle is not readily reconstructed. Moreover, the human body contains more than 600 striated muscles that are widely distributed, and these muscles control vital functions such as respiration, chewing and swallowing for food intake, and blood circulation. Muscles also regulate sugar and lipid metabolism, support the vertebrae, avoiding collapse or curving of the spinal column, and allow locomotion. All these functions are compromised in inherited muscular dystrophies, because both cardiac and skeletal muscles are often simultaneously affected. So far, the majority of preclinical and clinical therapeutic trials aimed at harnessing the regenerative properties of stem cells have focused on restoring just one tissue, heart or skeletal muscle. Therefore, a strategy that would allow for the combined regeneration of both striated muscle types would constitute a major advancement in the treatment of muscular dystrophies.

1.What is being aimed at controlling regenerative properties of stem cells?

a, pre clinical and clinical therapeutic trials.

b, pre clinical and majority of clinical therapeutic trials.

c, Most of the preclinical and clinical therapeutic trials .

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