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Post by imz72 on Dec 3, 2016 21:59:38 GMT
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Post by iamaverb on Dec 7, 2016 17:32:21 GMT
"Gene editing technologies have been developed to correct disease-causing genetic mutations, functionally replace and/or knock-out expression of dysfunctional genes. Nuclease-based methodologies for editing the genome dominate the field of gene editing" "Multiplexing capability as well as an observed increased targeting efficiency of CRISPR/Cas over TALENs capability has made CRISPR technology perhaps the most popular choice for gene editing" "Proof of principle studies includes a report where Crispr/Cas gene editing was used to correct the mutation of the β-globin gene in iPSCs from a β-thalassemia patient [109]. These corrected iPSCs displayed improved differentiation capacity into various types of hematopoietic progenitors and may be one day used as a source of autologous hematopoietic stem cells for transplantation and repopulation of the hematopoietic system. Similarly, Crispr/Cas9 was used to correct a mutation in the gene encoding the RP GTPase regulator in iPSCs derived from a patient with X-linked RP [127]. These corrected cells could in principle be differentiated into photoreceptors or their progenitors and used in cell replacement strategies for RP patients." It is interesting that Dr. Lanza and Kimbrel wrote a lot about the benefits derived by using the CRISPR-Cas9 PSC-based gene editing on PCS cells. I have a thread on CRISPR-Cas9 Gene Editing in the Non Stem Cell thread. Gene Editing
This is the next frontier. |
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Post by iamaverb on Dec 11, 2016 20:55:28 GMT
Genome Editing of Pluripotent Stem Cells could forecast the next decade of biotech
"It is extremely rare for a single experiment to be so impactful and timely that it shapes and forecasts the experiments of the next decade. Researchers review how two such experiments—the generation of human induced pluripotent stem cells (iPSCs) and the development of CRISPR/Cas9 technology—have fundamentally reshaped our approach to biomedical research, stem cell biology, and human genetics. They highlight the previous knowledge that iPSC and CRISPR/Cas9 technologies were built on as this groundwork demonstrated the need for solutions and the benefits that these technologies provided and set the stage for their success."
I will be interesting if Astellas begins licensing a CRISPR-Cas9 system to complement Lanza's stem cell research
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