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Nature Biotechnology

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https://www.readbyqxmd.com/read/28191903/efficient-genome-editing-in-the-mouse-brain-by-local-delivery-of-engineered-cas9-ribonucleoprotein-complexes
#1
Brett T Staahl, Madhurima Benekareddy, Claire Coulon-Bainier, Ashwin A Banfal, Stephen N Floor, Jennifer K Sabo, Cole Urnes, Gabriela Acevedo Munares, Anirvan Ghosh, Jennifer A Doudna
We demonstrate editing of post-mitotic neurons in the adult mouse brain following injection of Cas9 ribonucleoprotein (RNP) complexes in the hippocampus, striatum and cortex. Engineered variants of Cas9 with multiple SV40 nuclear localization sequences enabled a tenfold increase in the efficiency of neuronal editing in vivo. These advances indicate the potential of genome editing in the brain to correct or inactivate the underlying genetic causes of neurological diseases.
February 13, 2017: Nature Biotechnology
https://www.readbyqxmd.com/read/28191902/dynamic-regulation-of-metabolic-flux-in-engineered-bacteria-using-a-pathway-independent-quorum-sensing-circuit
#2
Apoorv Gupta, Irene M Brockman Reizman, Christopher R Reisch, Kristala L J Prather
Metabolic engineering of microorganisms to produce desirable products on an industrial scale can result in unbalanced cellular metabolic networks that reduce productivity and yield. Metabolic fluxes can be rebalanced using dynamic pathway regulation, but few broadly applicable tools are available to achieve this. We present a pathway-independent genetic control module that can be used to dynamically regulate the expression of target genes. We apply our module to identify the optimal point to redirect glycolytic flux into heterologous engineered pathways in Escherichia coli, resulting in titers of myo-inositol increased 5...
February 13, 2017: Nature Biotechnology
https://www.readbyqxmd.com/read/28191901/increasing-the-genome-targeting-scope-and-precision-of-base-editing-with-engineered-cas9-cytidine-deaminase-fusions
#3
Y Bill Kim, Alexis C Komor, Jonathan M Levy, Michael S Packer, Kevin T Zhao, David R Liu
Base editing induces single-nucleotide changes in the DNA of living cells using a fusion protein containing a catalytically defective Streptococcus pyogenes Cas9, a cytidine deaminase, and an inhibitor of base excision repair. This genome editing approach has the advantage that it does not require formation of double-stranded DNA breaks or provision of a donor DNA template. Here we report the development of five C to T (or G to A) base editors that use natural and engineered Cas9 variants with different protospacer-adjacent motif (PAM) specificities to expand the number of sites that can be targeted by base editing 2...
February 13, 2017: Nature Biotechnology
https://www.readbyqxmd.com/read/28165476/gene-therapy-restores-auditory-and-vestibular-function-in-a-mouse-model-of-usher-syndrome-type-1c
#4
Bifeng Pan, Charles Askew, Alice Galvin, Selena Heman-Ackah, Yukako Asai, Artur A Indzhykulian, Francine M Jodelka, Michelle L Hastings, Jennifer J Lentz, Luk H Vandenberghe, Jeffrey R Holt, Gwenaëlle S Géléoc
Because there are currently no biological treatments for hearing loss, we sought to advance gene therapy approaches to treat genetic deafness. We focused on Usher syndrome, a devastating genetic disorder that causes blindness, balance disorders and profound deafness, and studied a knock-in mouse model, Ush1c c.216G>A, for Usher syndrome type IC (USH1C). As restoration of complex auditory and balance function is likely to require gene delivery systems that target auditory and vestibular sensory cells with high efficiency, we delivered wild-type Ush1c into the inner ear of Ush1c c...
February 6, 2017: Nature Biotechnology
https://www.readbyqxmd.com/read/28165475/a-synthetic-aav-vector-enables-safe-and-efficient-gene-transfer-to-the-mammalian-inner-ear
#5
Lukas D Landegger, Bifeng Pan, Charles Askew, Sarah J Wassmer, Sarah D Gluck, Alice Galvin, Ruth Taylor, Andrew Forge, Konstantina M Stankovic, Jeffrey R Holt, Luk H Vandenberghe
Efforts to develop gene therapies for hearing loss have been hampered by the lack of safe, efficient, and clinically relevant delivery modalities. Here we demonstrate the safety and efficiency of Anc80L65, a rationally designed synthetic vector, for transgene delivery to the mouse cochlea. Ex vivo transduction of mouse organotypic explants identified Anc80L65 from a set of other adeno-associated virus (AAV) vectors as a potent vector for the cochlear cell targets. Round window membrane injection resulted in highly efficient transduction of inner and outer hair cells in mice, a substantial improvement over conventional AAV vectors...
February 6, 2017: Nature Biotechnology
https://www.readbyqxmd.com/read/28178263/novartis-snaps-up-selexys-for-sickle-cell
#6
Chris Morrison
No abstract text is available yet for this article.
February 8, 2017: Nature Biotechnology
https://www.readbyqxmd.com/read/28178262/correction
#7
(no author information available yet)
No abstract text is available yet for this article.
February 8, 2017: Nature Biotechnology
https://www.readbyqxmd.com/read/28178261/the-problem-with-neoantigen-prediction
#8
EDITORIAL
(no author information available yet)
No abstract text is available yet for this article.
February 8, 2017: Nature Biotechnology
https://www.readbyqxmd.com/read/28178260/fourth-quarter-biotech-job-picture
#9
Michael Francisco
No abstract text is available yet for this article.
February 8, 2017: Nature Biotechnology
https://www.readbyqxmd.com/read/28178259/roche-breaks-with-pacbio-opts-for-genia
#10
(no author information available yet)
No abstract text is available yet for this article.
February 8, 2017: Nature Biotechnology
https://www.readbyqxmd.com/read/28178258/industrializing-rare-disease-therapy-discovery-and-development
#11
Sean Ekins
No abstract text is available yet for this article.
February 8, 2017: Nature Biotechnology
https://www.readbyqxmd.com/read/28178257/drug-pipeline-4q16
#12
Laura DeFrancesco
No abstract text is available yet for this article.
February 8, 2017: Nature Biotechnology
https://www.readbyqxmd.com/read/28178256/jun-wang-s-icarbonx-heads-consortium-using-ai-in-health-and-wellness
#13
David Cyranoski
No abstract text is available yet for this article.
February 8, 2017: Nature Biotechnology
https://www.readbyqxmd.com/read/28178255/ge-to-boost-yields-with-synpromics-synthetic-promoters
#14
(no author information available yet)
No abstract text is available yet for this article.
February 8, 2017: Nature Biotechnology
https://www.readbyqxmd.com/read/28178254/aacr-makes-public-19-000-cancer-genomes
#15
(no author information available yet)
No abstract text is available yet for this article.
February 8, 2017: Nature Biotechnology
https://www.readbyqxmd.com/read/28178253/core-promoters-across-the-genome
#16
Nevena Cvetesic, Boris Lenhard
No abstract text is available yet for this article.
February 8, 2017: Nature Biotechnology
https://www.readbyqxmd.com/read/28178252/situating-brain-regions-among-patent-rights-and-moral-risks
#17
Dylan Roskams-Edris, Stacey Anderson-Redick, Zelma H Kiss, Judy Illes
No abstract text is available yet for this article.
February 8, 2017: Nature Biotechnology
https://www.readbyqxmd.com/read/28178251/recent-patents-in-single-cell-analysis-and-microfluidics
#18
(no author information available yet)
No abstract text is available yet for this article.
February 8, 2017: Nature Biotechnology
https://www.readbyqxmd.com/read/28178250/spinal-muscular-atrophy-approval-boosts-antisense-drugs
#19
Elie Dolgin
No abstract text is available yet for this article.
February 8, 2017: Nature Biotechnology
https://www.readbyqxmd.com/read/28178248/stretching-sensors-by-confinement
#20
Irene Jarchum
No abstract text is available yet for this article.
February 8, 2017: Nature Biotechnology
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