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Researchers are using the CRISPR-Cas9 system to genetically engineer organisms i

ID: 301897 • Letter: R

Question

Researchers are using the CRISPR-Cas9 system to genetically engineer organisms in order to study gene function and to attempt to help patients with genetic diseases. In 2016, a research team used the CRISPR-Cas9 system to genetically alter pig zygotes so that they would have muscular dystrophy, a type of genetic disease that results in muscle wasting and loss of muscle mass. This disease often results from a lack of the protein dystrophin in skeletal muscle tissue. Researchers sometimes develop animal models of diseases so that they can develop therapies to treat the humarn version of the disease. The researchers compared the molecular biology and physiology of two types of pigs: healthy, normal pigs (WT) and those that were modified when they were zygotes with the CRISPR-Cas9 system (CRISPR). The data below show the dystrophin levels in various tissues from both types of pigs. 2- 1.5 0.5 0 1 Skeletal musde Heart Intestine Source: Adapted from Yu, H. H Zhao, Qing, Y B Pan w R J a, ?.? Zhao H Y Huang X X et a 2016 Po in z o e injection ith a 9/sgRI muscle dystrophy. International Journal of Molecular Sciences, 17(10): 1668. MDPI AG. Retrieved from http:lldx.doi.org/10.3390ijms17101668 result in ONO-modified pig with In this study, what would have been true of the guide RNA that was used in the CRISPR-Cas9 system? O The guide RNA helped target the Cas9 enzyme to the dystrophin protein. O The guide RNA was injected into skeletal muscle cells, heart cells, and intestinal cells. The guide RNA was complementary in sequence to a region of the dystrophin gene.

Explanation / Answer

Answer: In CRISPER/Cas9 system of gene editing, The guide RNA and Cas9 form a complex to identify the target sequence with high specificity. Cas9 remains inactive till the guide RNA binds with it and form a complex.

Therefore, Option (1) i.e. the guide RNA helped target the Cas9 enzyme to the dystrophin pritein is TRUE.

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