Spinal Muscular Atrophy (SMA) Genetics Quiz

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What is the cause of SMA?

Recessive mutations of the SMN1 gene

What is the function of the SMN2 gene in SMA patients?

Capable of producing low levels of full-length SMN protein

What is the typical outcome for affected infants with severe muscle weakness?

Die before reaching two years of age

What are some of the therapeutic targets for SMA treatment?

Activate SMN2 gene expression, modulate splicing of SMN2 preRNAs, stabilize SMN protein, replace SMN1, provide neuroprotective support, and transplant neural cells Signup and view all the answers

What is the current status of treatments for SMA patients?

No treatments currently exist Signup and view all the answers

What has resulted in increased drug development efforts for SMA in the past decade?

Identification of therapeutic targets and the development of suitable animal models for preclinical testing Signup and view all the answers

Spinal Muscular Atrophy (SMA) Genetics Quiz

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Questions and Answers

What is the cause of SMA?

What is the function of the SMN2 gene in SMA patients?

What is the typical outcome for affected infants with severe muscle weakness?

What are some of the therapeutic targets for SMA treatment?

What is the current status of treatments for SMA patients?

What has resulted in increased drug development efforts for SMA in the past decade?

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