Bringing you the latest cutting-edge research and commentary in bioscience.

Cell & Gene Therapy Insights

Cell & Gene Therapy Insights

Issue Vol 3 Issue 1

Editorial

In vivo genome editing as a therapeutic strategy for treatment of inherited retinal dystrophies: how close are we to realization?

Spotlight Article

Editorial

Translation and Application of Gene Editing

Knut Stieger Gene Editing Technologies Inherited retinal dystrophies are a heterogeneous group of genetic diseases for which currently no effective treatment strategies exist. Gene editing as a therapeutic strategy for these diseases are currently being studied and this editorial provides an overview of the various in vivo gene editing approaches developed for the treatment of […]

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Advances & challenges of using CRISPR-Cas9 gene editing for treating Duchenne muscular dystrophy

Spotlight Article

Editorial

Translation and Application of Gene Editing

Dominique L Ouellet, Benjamin Duchêne, Jean-Paul Iyombe-Engembe & Jacques P Tremblay Gene Editing Technologies Genome editing with the CRISPR/Cas9 system has recently been used as a promising strategy to treat Duchenne muscular dystrophy (DMD) both in vitro and in vivo. This editorial provides an overview of the advances and challenges associated with translating this gene […]

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Commercial Insights

Cell & Gene Therapy Commercial Insight – January 2017

Commercial Insights

Translation and Application of Gene Editing

Mark Curtis & Richard Philipson Providing a critical overview of the sector’s commercial developments – M&As, licensing agreements & collaborations, financial results, IPOs and clinical/regulatory updates, with commentary from our Expert Contributors. CELL THERAPY: It was a busy start to 2017 with multiple new immunotherapy products en route to the clinic. Kite submitted an IND […]

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Interview

The scope and challenges of visualizing CRISPR-Cas9 gene editing in real time

Spotlight Article

Interview

Translation and Application of Gene Editing

Gene Editing Technologies Dr. Krishanu Saha Dr. Krishanu Saha is an Assistant Professor in the Department of Biomedical Engineering at the University of Wisconsin-Madison. He is also a member of the Wisconsin Institute for Discovery in the bio-nanocomposite tissue engineering scaffolds theme. Prior to his arrival in Madison, Dr. Saha studied Chemical Engineering at Cornell […]

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Gene editing of peripheral blood T cells for cancer immunotherapy

Spotlight Article

Interview

Translation and Application of Gene Editing

Gene Editing Technologies Dr Avery Posey Dr Avery Posey is an immunologist with training in bioinformatics, biochemistry, and molecular and developmental genetics. He completed his postdoctoral training in the Center for Cellular Immunotherapies at the University of Pennsylvania with Dr Carl H. June investigating novel CAR T cells for solid tumors; CAR T cell costimulation, […]

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Expert Insight

Repurposing CRISPR-Cas systems as DNA-based smart antimicrobials

Spotlight Article

Expert Insight

Translation and Application of Gene Editing

Rodolphe Barrangou & David G Ousterout Gene Editing Technologies The molecular machines from CRISPR-Cas immune systems provide versatile platforms for programmable targeting of DNA, which is commonly used to edit eukaryotic genomes, transcriptomes and epigenomes. Recently, CRISPR-Cas systems have been repurposed as specific antimicrobials, given their ability to selectively target and degrade bacterial DNA. Endogenous […]

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Current gene editing strategies for the mucopolysaccharidoses

Spotlight Article

Expert Insight

Translation and Application of Gene Editing

Mark J Osborn, Dok Hyun Yoon, Beom Hee Lee, Chong Jai Kim & Jakub Tolar Gene Editing Technologies The mucopolysaccharidoses are one of the subclassifications of a group of metabolic disorders termed lysosomal storage disease. They occur from mutations to genes encoding enzymes responsible for glycosaminoglycan degradation. The ramifications of glycosaminoglycan accumulation are far reaching […]

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Recent advances in the use of ZFN-mediated gene editing for human gene therapy

Spotlight Article

Expert Insight

Translation and Application of Gene Editing

Srinivasan Chandrasegaran Gene Editing Technologies Targeted genome editing with programmable nucleases has revolutionized biomedical research. The ability to make site-specific modifications to the human genome, has invoked a paradigm shift in gene therapy. Using gene editing technologies, the sequence in the human genome can now be precisely engineered to achieve a therapeutic effect. Zinc finger […]

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