Genetics help Duchenne muscular dystrophy (DMD) is an X-linked recessive genetic

Question

Genetics help Duchenne muscular dystrophy (DMD) is an X-linked recessive genetic disease caused by mutations in the gene that encodes dystrophin, a large protein that plays an important role in the development of normal muscle fibers. The dystrophin gene is immense, spanning 2.5 million base pairs, and includes 79 exons and 78 introns Many of the mutations that cause DMD produce premature stop codons, which bring protein synthesis to a halt, resulting in a greatly shortened and nonfunctional form of dystrophin. Some geneticists have proposed treating DMD patients by introducing small RNA molecules that cause the spliceosome to skip the exon containing the stop codon. The introduction of the small RNAs will produce a protein that is somewhat shortened because an exon is skipped and some amino acids are missing, but it may still result in a protein that has some function. The small RNAs, antisense RNAs, used for exon skipping are complementary to bases in the pre-mRNA, which will prevent 306:1796-1799) associating of spliceosome for intron removal. (A. Goyenvalle et al., 2004. Science In order to skip the mutated exon and potentially treat DMD, select the best antisense RNA targets. a 3' splice site of the intron downstream of the exon to be skipped a 5 splice site of the intron downstream of the exon to be skipped a 3 splice site of the intron upstream of the exon to be skipped a 5. splice site of the intron upstream of the exon to be skipped a branch point of the intron upstream of the exon to be skipped

Explanation / Answer

Stop codon is present downstream at the 3' end. The mutated exon is flanked by introns at the 5' end and at the 3' end. To join the normal exons on two sides of this mutated exon and to splice out the mutated exon, the 3' splice site of the upstream introns and 5' splice site of the downstream introns should be skipped. The branch-point sequences located upstream at the exon-intron boundary is another site to be skipped.

So, answer 2,3 and 5 are correct.

Related Questions

Navigate

• Browse (All)
• Browse G
• Subjects

---

---

Integrity-first tutoring: explanations and feedback only — we do not complete graded work. Learn more.