Gene therapy is an insertion of functional gene in the location of dysfunctional gene or neighboring to it. we have just last week the first report of use of CRISPR technology to alter a human embryo — but these were not permitted to develop beyond a few days. So it demonstrated that, yes, you can use CRISPR to edit DNA of a human embryo — but you can’t call it a “successful gene therapy” yet because this was an “in the lab” technique, not one with a viable human patient.
CRISPER is one laboratory method of altering the DNA and can be used as a tool for gene therapy, whereas gene therapy is a complete domain of treating genetic disorders by using number of techniques of genetic manipulation...
Gene therapy is a broad strategy for curing or treating disease by modifying, supplying or blocking genes or gene products that cause a condition either by their presence or absence. The treatments typically involve delivery of synthetically manufactured nucleic acid polymers, which are relatively expensive to manufacture. Early attempts caused unintended outcomes that inhibited progress. Gene therapy is any attempt to edit genes to cure some disease.
CRISPR/Cas9 is a method of genome editing. CRISPR used for gene therapy (or for any other gene editing attempts), and gene therapy could be done with any of several other methods.
Mushtak here i attach one commentary may be it will help you.
Find this attachment.
CRISPER is one laboratory method of altering the DNA and can be used as a tool for gene therapy, whereas gene therapy is a complete domain of treating genetic disorders by using number of techniques of genetic manipulation...
In short, CRISPR/Cas9 is a molecular tool, which can be used for 'gene therapy'. CRISPR/Cas9 is able to deliver a correct gene into human genome to fix a defect gene.
Dear Mushtak T. S. Al-Ouqaili,
For your statement: "So it demonstrated that, yes, you can use CRISPR to edit DNA of a human embryo — but you can’t call it a “successful gene therapy” yet because this was an “in the lab” technique, not one with a viable human patient."
I have two comments:
1. Fix a defect gene from as early as an embroy, so the developed adult can be healthy, is also considered a successful gene therapy.
2. Use CRISPR/Cas9 technology on human patiants are under way (2018). The first trial will be on 'sickle cell disease'. Read this article:
CRISPR to Debut in Clinical Trials The first industry-sponsored CRISPR therapy is slated to be tested in humans in 2018. By Diana Kwon | December 14, 2017
"CRISPR Therapeutics submitted an application to European regulatory authorities seeking permission to begin clinical trials for CTX001, an investigational CRISPR treatment for patients with sickle cell disease and β thalassemia."
https://www.the-scientist.com/?articles.view/articleNo/51174/title/CRISPR-to-Debut-in-Clinical-Trials/&utm_content=64537164&utm_medium=social&utm_source=facebook/
Hi Mushtak T. S. Al-Ouqaili,
For some diseases it is better to fix a defect gene as soon as at 'embryo' stage (such as heart disease, muscle disease,....), so the new-born baby wouldn't carry the disease. Otherwise, it might be too late.
Here is a report from CNN about scientists using CRISPR to correct a heart disease gene from human embryo. (https://www.cnn.com/2017/08/02/health/crispr-human-embryos-gene-editing-study/index.html)
Statement in the CNN report from a researcher: "...........So when the baby is born, all the cells do not have the mutation anymore. ... This study, it shows that we can correct the embryo and then, after the division, all the cells are corrected, so there's not what we call mosaicism," said Belmonte, who is also a member of the National Academies of Sciences, Engineering and Medicine's committee on human gene editing............"
CRISPR is Clustered Regularly Interspaced Short Palindromic Repeats. These are the hallmark of a bacterial defense system that forms the basis for CRISPR-Cas9 genome editing technology (a technique).
Gene therapy is an application of also this technology.
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