Harvard University: Timeline of key events

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J. S. Gootenberg, O.O. Abudayyeh, J. W. Lee, et al, 'Nucleic acid detection with CRISPR-Cas13a/C2c2', Science, 13 April 2017, eaam9321, DOI: 10.1126/science.aam9321 2017-04-13T00:00:00+0000L. Xu, et al, 'Trispecific broadly neutralizing HIV antibodies mediate potent SHIV protection in macaques', Science, 20 September 2017, Science, 20 Sep 2017, eaan8630, DOI: 0.1126/science.aan86302017-09-20T00:00:00+0000Total of 17 boys treated in clinical trial, of which 15 showed marked improvement. Treatment used a modified form of HIV as the vector for infusing corrective genes to generate glial cells. F. Eichler, C. Duncan etl al, 'Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy', NEJM, DOI: 10.1056/NEJMoa17005542017-10-04T00:00:00+0000N.M. Gaudelli, A. Komor, H. A. Rees, M. S. Packer, A. H. Badran, D. I. Bryson, D. R. Liu, 'Programmable base editing of A-T to G-C in genomic DNA without DNA cleavage', Nature (2017), doi:10.1038/nature24644. 2017-10-25T00:00:00+0000Twenty-two patients were treated in the trial over 42 months in six centres around the world. The treatment consisted of taking immature stem cells from the patient's bone marrow and using a harmless virus to infect the cells with a copy of the normal globin gene. The new genetically altered cells were then reintroduced into the patient's bloodstream after their marrow had been cleared of diseased cells using chemotherapy. Nine out of the twenty-two patients severe beta thalassemia were able to cut down on the number of transfusions they needed by 74%, with three of them no longer needing any transfusions at all. The same was true of the twelve out of thirteen patients with less severe thalassemia. A A Thompson et al, 'Gene therapy in patients with transfusion-dependent beta thalassemia', New England Journal of Medicine, 378 (2018), 1479-93.2018-04-19T00:00:00+0000The advantage of the technique is it enables scientists to disable specific genes and correct harmful mutations by providing cells with fresh DNA strands with to repair the cut. Unlike Crispr-Cas9, which chops target DNA in half, prime editing nicks it and then writes a new section of DNA into the specified region. The new procedure was published in AW Anzalone et al, 'Search-and-replace genome editing without double-strand breaks or donor DNA', Nature (2019), https://doi.org/10.1038/s41586-019-1711-42019-10-21T00:00:00+0000
Date Event People Places Sciences
13 Apr 2017CRISPR shown to be sensitive diagnostic tool for detecting single target of DNA or RNA moleculeAbudayyeh, Bhattacharyya, Collins, Daringe, Donghia, Dy, Essletzbichler, Freije, Hung, Joung, Koonin, Lee, Livny, Myhrvold, Regev, Sabeti, Gootenberg, Verdine, ZhangBroad Institute, Massachusetts Institute of Technology, Harvard University, Howard Hughes Medical Institute CRISPR-Cas9, Gene editing
20 Sep 2017Combination of three monoclonal antibodies reported to protect monkeys from HIV Xu, Pegu, Rao, Doria-Rose, Beninga, McKee, Lord, Wei, Deng, Louder, Schmidt, Mankoff, Wu, Asokan, Beil, Lange, Leuschner, Kruip, Sendak, Kwon, Zhou, Chen, Bailer, Wang, Choe, Tartaglia, Barouch, O’Dell, Todd, Burton, Roederer, Connors, Koup, Kwong, YangSanhofi, National Institute of Allergy and Infectious Disease, Harvard University, Massachusetts Institute of Technology, Scripps Research Institute, Monoclonal antibodies, Infectious diseases
4 Oct 2017Gene therapy shown in clinical trials to halt progression of adrenoleukodystrophy, a fatal brain disease inherited by boys Eichler, Duncan, WilliamsHarvard University, Bluebird Bio, Boston Children’s Hospital Stem cells, Gene therapy
25 Oct 2017Base editing improvements announced for CRISPR technique, providing means to change individual chemical letters of DNA without need to cleave DNAGaudelli, Komor, Rees, Packer, Badran, Bryson, LiuMassachusetts Institute of Technology, Harvard UniversityCRISPR-Cas9, Gene editing
19 Apr 2018Gene therapy shown to be promising treatment in clinical trials for beta thalassemiaThompson, Walters, Kwiatkowski, Rasko, Ribeil, Hongeng, Magrin, Schiller, Payen, Smeraro, Moshous, LefrerNorth Western University, University of California San Francisco, University of California Los Angeles, University of Sydney, University of Paris, Harvard University, Mahidol University, German Cancer Research CentreGene therapy
21 Oct 2019New DNA editing technique called 'prime editing' publishedAnzalone, Randolph, Davis, Sousa, Koblan, Levy, Chen, Wilson, Newby, Ranguram, LiuMassachusetts Institute of Technology, Harvard UniversityCrispr-Cas9, Gene editing,

13 Apr 2017

CRISPR shown to be sensitive diagnostic tool for detecting single target of DNA or RNA molecule

20 Sep 2017

Combination of three monoclonal antibodies reported to protect monkeys from HIV

4 Oct 2017

Gene therapy shown in clinical trials to halt progression of adrenoleukodystrophy, a fatal brain disease inherited by boys

25 Oct 2017

Base editing improvements announced for CRISPR technique, providing means to change individual chemical letters of DNA without need to cleave DNA

19 Apr 2018

Gene therapy shown to be promising treatment in clinical trials for beta thalassemia

21 Oct 2019

New DNA editing technique called 'prime editing' published