AMAZING developments in treating sickle cell disease with genetic therapies ...
What is sickle cell disease?
Sickle cell anemia
- https://www.mayoclinic.org/diseases-conditions/sickle-cell-anemia/symptoms-causes/syc-20355876
(and below)
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It affects more than 2 million people worldwide, and about 100,000 people in the USA.
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Brainstorming-wise, in what ways in a SINGLE realistic virtual earth for STEM, for CRISPR, and with machine learning, could faculty and graduate students at World Univ and Sch and around the world, help contribute to such fascinating, and remarkable, successful genetic therapy developments as for sickle cell anemia? Longevity too ... https://wiki.worlduniversityandschool.org/wiki/Longevity (https://wiki.worlduniversityandschool.org/wiki/Subjects) ?
And with regard to simulations into Google Poly (a library of virtual world objects) too?
searched on ...
'sickle cell anemia gene therapy avatar bots computer simulations' ...
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At World Univ & Sch ...
Genetics -
https://wiki.worlduniversityandschool.org/wiki/Genetics
Biomedical Engineering -
https://wiki.worlduniversityandschool.org/wiki/Biomedical_Engineering
Biological Engineering -
https://wiki.worlduniversityandschool.org/wiki/Biological_Engineering
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Computer model could help test new sickle cell drugs
- August 22, 2019
- Brown University
- A new computer model that captures the dynamics of the red blood cell sickling process could help in evaluating drugs for treating sickle cell disease.
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Computer models provide new understanding of sickle cell disease
https://phys.org/news/2017-07-sickle-cell-disease.html* *
Sickle Cell Disease
Also known as Sickle Cell Anemia, Hemoglobin S or SS disease, Sickling disorder due to hemoglobin S
- https://www.nhlbi.nih.gov/health-topics/sickle-cell-disease*
Sickle cell disease
- https://ghr.nlm.nih.gov/condition/sickle-cell-disease#statistics
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Patients respond to gene therapy treatment for sickle cell anemia
https://youtu.be/qNxwq10kXXk
https://www.youtube.com/watch?v=qNxwq10kXXk
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Gene Therapy for Sickle-Cell Anemia Looks Promising—but It’s Riddled With Controversy
By Shelly Fan - Dec 17, 2019
https://singularityhub.com/2019/12/17/gene-therapy-for-sickle-cell-anemia-looks-promising-but-its-riddled-with-controversy/
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NIH researchers create new viral vector for improved gene therapy in sickle cell disease
‘Forward-oriented’ design might boost treatment effectiveness and broaden use.
- https://www.nih.gov/news-events/news-releases/nih-researchers-create-new-viral-vector-improved-gene-therapy-sickle-cell-disease* *
Gene therapy[edit]
In 2001, sickle cell disease reportedly had been successfully treated in mice using gene therapy.[125][126] The researchers used a viral vector to make the mice—which have essentially the same defect that causes human sickle cell disease—express production of fetal haemoglobin (HbF), which an individual normally ceases to produce shortly after birth. In humans, using hydroxyurea to stimulate the production of HbF has been known to temporarily alleviate sickle cell disease symptoms. The researchers demonstrated that this gene therapy method is a more permanent way to increase therapeutic HbF production.[127]
Phase 1 clinical trials of gene therapy for sickle cell disease in humans were started in 2014. The clinical trials will assess the safety of lentiviral vector-modified bone marrow for adults with severe sickle cell disease.[128][129] As of 2018, however, no randomized controlled trials have been reported.[130] A case report for the first person treated was published in March 2017, with a few more people being treated since then.[131][132]
Gene editing platforms like CRISPR/Cas9 have been used to correct the disease-causing mutation in hematopoietic stem cells taken from a person with the condition.[133] In July 2019 the gene-editing tool CRISPR was used to edit bone marrow cells from a person with SCD to "turning on" the gene for fetal haemoglobin.[134]
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Diseases Treated by Gene Therapy
http://www.genetherapynet.com/JoomlaTest2/index.php?option=com_content&view=article&id=164:diseases-treated-with-gene-therapy-&catid=97:patient-information&Itemid=14Biotechnology / Genetic Engineering
2017 Was the Year of Gene-Therapy Breakthroughs
Gene-fixing treatments have now cured a number of patients with cancer and rare diseases.
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Gene Therapy in a Patient with Sickle Cell Disease
https://www.nejm.org/doi/full/10.1056/NEJMoa1609677 -
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This blog entry seeks to explore questions related to
"what role does sickle cell disease gene therapy play in genetic therapy revolution"? - potentially for large numbers of people.
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https://en.wikipedia.org/wiki/Blood_cell
https://en.wikipedia.org/wiki/Sickle_cell_disease
https://en.wikipedia.org/wiki/Congenital_dyserythropoietic_anemia
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