X linked recessive mutations in the dystrophin gene cause muscular dystrophy. The gene is enormous, over 1 million base pairs. Because it is that large, the gene can not be readily inserted into a virus for gene therapy. What other technique that we have discussed could you use to do gene therapy on this condition?

X linked recessive mutations in the dystrophin gene cause muscular dystrophy. The gene is enormous, over 1 million base pairs. Because it is that large, the gene can not be readily inserted into a virus for gene therapy. What other technique that we have discussed could you use to do gene therapy on this condition?

Biology (MindTap Course List)

11th Edition

ISBN:9781337392938

Author:Eldra Solomon, Charles Martin, Diana W. Martin, Linda R. Berg

Publisher:Eldra Solomon, Charles Martin, Diana W. Martin, Linda R. Berg

Chapter16: Human Genetics And The Human Genome

Section16.2: Abnormalities In Chromosome Number And Structure

Problem 3C

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Genes with highly similar sequence are often located adjacent one another in the genome. Gene duplication commonly arises from errors in replication. When the organization of such adjacent genes is in an inverted orientation, this can reduce the expression of other genes that have similar sequence and are located on other chromosomes. Explain the mechanism of how this generally occurs. i need a detailed explanation, please. I need to know what the mechanism is. How it happens and why. Please. Thanks
I need help because i don't get it. Can you help me to explain to me, please?
In contrast with the genomic manipulations of animals and plants described in this chapter, human genetherapy is directed specifically at altering the genomes of somatic cells rather than germ-line cells.Why couldn’t or wouldn’t medical scientists try to alter the genome of human germ-line cells?
Shown below are the maps of a series of rII− deletion strains (1–5). The deleted region is indicated as (......) and the intact region as ______. 1 ___________(...........)_______________ 2 _________________(...........)_________ 3 (.....................)_____________________ 4 ________________________(................) 5 _____(..........)______________________ rII− phage strains A-E have point mutations in the rII region. E.coli K(λ) cells are coinfected with one phage that has a deletion and one phage that has a point mutation. The presence of wild-type progeny phage is assessed by the presence (+) or absence (o) of plaques. 1 2 3 4 5 A + o + + + B o + + + + C + + + o + D + + o + + E + + o + o A) Indicate the order of the point mutations in the rII region. A) CADBE B) DEBAC C) BADCE D) ABDEC E) CEADB B) The test described here is a recombination test. Explain…
Genes with highly similar sequence are often located adjacent one another in the genome. Gene duplication commonly arises from errors in replication. When the organization of such adjacent genes is in an inverted orientation, this can reduce the expression of other genes that have similar sequence and are located on other chromosomes. Explain the mechanism of how this generally occurs. Please state the answer in details: what is the mechanism? How it happens? Why this happens? When it happens? And every other necessary information.
this is what i have said about this image so far, what else can be said aswell including the raw count column. " Interpreting the results of an RNA-Seq analysis is pivotal in understanding the underlying genetic mechanisms of diseases such as breast cancer. In this analysis, Figure 1 provides comprehensive data on differentially expressed genes associated with breast cancer. By delving into the provided information, we can gain valuable insights into the molecular landscape of this disease. First focus is on the gene with the highest fold change, EYA4, situated on chromosome 6. With a staggering fold change of 3604.4176, EYA4 exhibits an unprecedented level of overexpression in cancerous cells compared to normal cells. This profound alteration suggests a pivotal role for EYA4 in breast cancer pathogenesis. The log2 fold change of 11.81555 further emphasizes the magnitude of this difference in gene expression. Statistical significance is evident, with an exceptionally low p-value of…
1). In the absence of this enzyme, a substance called ceroid lipofuscin accumulates in lysosomes in the brain, resulting in seizures, blindness, decline in cognitive function and motor skills, dementia, and death by the late teens or early 20’s. The TPP1 gene is 6695 bp in length. Think about the characteristics of Batten disease, and then suggest an approach to gene therapy that might be effective for this specific genetic disorder. You may assume that your research team is working in the U.S. and your research is funded by a grant from the National Institutes of Health (NIH). Please EXCLUDE the use of CRISPR from consideration. A. Will you use germline or somatic cell gene therapy? Please NAME and DEFINE the form of gene therapy selected, then explain WHY this is the most appropriate choice.
1). In the absence of this enzyme, a substance called ceroid lipofuscin accumulates in lysosomes in the brain, resulting in seizures, blindness, decline in cognitive function and motor skills, dementia, and death by the late teens or early 20’s. The TPP1 gene is 6695 bp in length. Think about the characteristics of Batten disease, and then suggest an approach to gene therapy that might be effective for this specific genetic disorder. You may assume that your research team is working in the U.S. and your research is funded by a grant from the National Institutes of Health (NIH). Other scientists have suggested that it might be possible to use CRISPR to treat this genetic disorder in affected individuals. (i) First, what is CRISPR? (BRIEFLY describe what it is and how it works). (ii) Briefly describe how CRISPR could be utilized in treating genetic conditions such as Batten disease.
Discuss the following types of mutations, with reference to specific genetic disorders: i) Chromosomal deletion; ii) Reciprocal translocation; and iii) Haploinsufficiency
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Look at Figure 17-22 and state which bands are missing in the cri du chat deletion.
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