know that CRISPR-Cas9 technology works in both cells and germ cells and in both primate and human embryos.” George Church (2015) Encourage the innovators Nature 528, 3 December, Page S7 is no technical reason why gene therapy could not be “There deployed to alter the human germ line” already know that germline-editing is unlikely to cause “We dangerous, unforeseen mutations” far CRISPR-Cas9 seems capable of less than 1 error in 300 “so trillion base pairs, and techniques to reduce these off-target effects using CRISPR pairs might cut this by many factors of ten” Michael Eisenstein (2015) Closing the door on HIV Nature 528, 3 December, Page S8-S9 Virginia Gewin (2015) Expanding possibilities Nature 528, 3 December, Page S10-S11 late 2013, Hans Clevers isolated intestinal stem cells from two “In children with cystic fibrosis He used these stem cells to grow gut … tissue…. And introduced a healthy version of the gene that is disrupted in people with cystic fibrosis The results were impressive; the faulty … gene was corrected in about half of the miniguts that Clevers tested. “ consensus is that monogenc diseases those involving only one “The – gene are the low lying fruit of the field” – hurdle is so substantial that researchers are trying to work “Thedelivery around it, rather than over come it.” Easiest are problems with the bone marrow including HIV, sickle cell anemia, disorders and genetic diseases affecting the eye. Also easier are diseases in which the patient has one mutant gene and one healthy gene where witching off the mutant gene solves the problem. Trickier is inserting a corrected copy of the gene in place of the defective copy.
know that CRISPR-Cas9 technology works in both cells and germ cells and in both primate and human embryos.” George Church (2015) Encourage the innovators Nature 528, 3 December, Page S7 “There is no technical reason why gene therapy could not be deployed to alter the human germ line” “We already know that germline-editing is unlikely to cause dangerous, unforeseen mutations” “so far CRISPR-Cas9 seems capable of less than 1 error in 300 trillion base pairs, and techniques to reduce these off-target effects using CRISPR pairs might cut this by many factors of ten” Michael Eisenstein (2015) Closing the door on HIV Nature 528, 3 December, Page S8-S9 Virginia Gewin (2015) Expanding possibilities Nature 528, 3 December, Page S10-S11 “In late 2013, Hans Clevers isolated intestinal stem cells from two children with cystic fibrosis … He used these stem cells to grow gut tissue…. And introduced a healthy version of the gene that is disrupted in people with cystic fibrosis … The results were impressive; the faulty gene was corrected in about half of the miniguts that Clevers tested. “ “The consensus is that monogenc diseases – those involving only one gene – are the low lying fruit of the field” “Thedelivery hurdle is so substantial that researchers are trying to work around it, rather than over come it.” Easiest are problems with the bone marrow including HIV, sickle cell anemia, disorders and genetic diseases affecting the eye. Also easier are diseases in which the patient has one mutant gene and one healthy gene where witching off the mutant gene solves the problem. Trickier is inserting a corrected copy of the gene in place of the defective copy.
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