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Genomics|Features|Gene Therapy

Gene Therapy & Genome Editing

Genome Editing: Scientific and Ethical Issues - June 18, 2015

Germline genome-editing research and its socioethical implications.
Ishii T et al. Trends Mol Med 2015 Jun 9.

Brave new genome
Lander ES NEJM 2015 Jun 3

CRISPR: the good, the bad and the unknown
Nature Special, May 2015

US science academies take on human-genome editing, by S Reardon, Nature News, May 18, 2015

Editing human germline cells sparks ethics debate
New methods to alter DNA bring science fiction closer to reality, T Hesman Saey, Science News, May 6

US government will not fund gene editing in human embryos, by P Brice, PHG Foundation, May 4

Bioethics. Embryo engineering study splits scientific community.
Kaiser J et al. Science 2015 May 1. (6234) 486-7

CRISPR germline editing reverberates through biotech community, Nature Biotechnology Blog, April 30, 2015

NIH reiterates ban on editing human embryo DNA
Agency issues statement after researchers delete a disease gene in nonviable zygotes, S. Reardon, Nature News, April 29, 2015

DNA editing in mouse embryos prevents disease, by Sara Reardon, Nature News, Apr 23

Technique aims to cut disease-causing mutations out of eggs and embryos, Science News, Apr 23

Editing human embryos: So this happened. by Carl Zimmer, National Geographic, Apr 22

Controversial world first genetic modification of human embryos, PHG Foundation, Apr 23

Breaking Mendelian inheritance with CRISPR-Cas
Lau E. Nature Reviews Genetics April 2015

Germline editing: time for discussion
Nature Medicine Editorial, April 8, 2015

Mini enzyme moves gene editing closer to the clinic
Discovery expands potential CRISPR toolbox for treating genetic diseases in humans. Heidi Ledford. Nature News, April 1, 2015

A prudent path forward for genomic engineering and germline gene modification.
Baltimore BD et al. Science 2015 Mar 19.

Scientists seek ban on method of editing the human genome, by Nicholas Wade, New York Times, Mar 19

CRISPR probes the inner workings of the genome in real time, by K Davis, NHGRI, February 2015

Therapeutic genome editing: prospects and challenges
David Benjamin, et al. Nature Medicine, February 5, 2015

Genome editing-based HIV therapies.
Gu WG. Trends Biotechnol. 2015 Jan 15.

Strategies for cloning and manipulating natural and synthetic chromosomes.
Karas BJ, et al. Chromosome Res. 2015 Jan 18

The impact of CRISPR-Cas9 on target identification and validation.
Moore JD. Drug Discov Today. 2015 Jan 5

Beyond traditional pharmacology: new tools and approaches.
Gurevich EV, Gurevich VV. Br J Pharmacol. 2015 Jan 9.

Unraveling the potential of CRISPR-Cas9 for gene therapy.
Barrangou R, May AP. Expert Opin Biol Ther. 2014 Dec 23:1-4

One-shot, one cure with genome editing for dyslipidemia.
Gupta RM. Circ Cardiovasc Genet. 2014 Dec;7(6):967-8

Correction of a genetic disease by CRISPR-Cas9-mediated gene editing in mouse spermatogonial stem cells.
Wu Y, et al. Cell Res. 2015 Jan;25(1):67-79

CRISPR-mediated genome editing of Plasmodium falciparum malaria parasites.
Lee MC, et al. Genome Med. 2014 Aug 26;6(8):63

Genome editing. The new frontier of genome engineering with CRISPR-Cas9.
Doudna JA, et al. Science. 2014 Nov 28;346(6213):1258096.

Genome editing for human gene therapy.
Meissner TB, et al. Methods Enzymol. 2014;546:273-95

Genome editing: a tool for research and therapy: targeted genome editing hits the clinic.
Lombardo A, et al. Nat Med. 2014 Oct;20(10):1101-3

Genome editing: a tool for research and therapy: towards a functional understanding of variants for molecular diagnostics using genome editing.
Tsai SQ, et al. Nat Med. 2014 Oct;20(10):1103-4

Call for debate on human genome editing, by Dr Philippa Brice, PHG Foundation, Jan 22

Gene Therapy & Gene Editing: Recent Insights - October 16, 2014

In hopes of fixing faulty genes, one scientist starts with the basics, by Joe Palca, National Public Radio, Oct 13

Advances in CRISPR genome-editing, PHG Foundation, Oct 7

CRISPR-Cas9 knockin mice for genome editing and cancer modeling.
Platt RJ, et al. Cell. 2014 Oct 9;159(2):440-55.

Sequence-specific antimicrobials using efficiently delivered RNA-guided nucleases
Citorik RJ, et al. Nat Biotechnol. 2014 Sep 21.

Exploiting CRISPR-Cas nucleases to produce sequence-specific antimicrobials.
Bikard D, et al. Nat Biotechnol. 2014 Oct 5.

RNA-directed gene editing specifically eradicates latent and prevents new HIV-1 infection
Hu W, et al.  PNAS, Jul 21, 2014.

Genome editing cuts out HIV
Grens K. The Scientist, Jul 21, 2014.

Gene researchers work to engineer HIV-resistant cells
Hampton T. JAMA, Jul 9, 2014.

Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV
Tebas P, et al. N Engl J Med. 2014 Mar 6;370(10):901-10.

Promising results from HIV gene editing therapy, PHG Foundation, Mar 10