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      Gene therapy: progress and predictions

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          Abstract

          The first clinical gene delivery, which involved insertion of a marker gene into lymphocytes from cancer patients, was published 25 years ago. In this review, we describe progress since then in gene therapy. Patients with some inherited single-gene defects can now be treated with their own bone marrow stem cells that have been engineered with a viral vector carrying the missing gene. Patients with inherited retinopathies and haemophilia B can also be treated by local or systemic injection of viral vectors. There are also a number of promising gene therapy approaches for cancer and infectious disease. We predict that the next 25 years will see improvements in safety, efficacy and manufacture of gene delivery vectors and introduction of gene-editing technologies to the clinic. Gene delivery may also prove a cost-effective method for the delivery of biological medicines.

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          Safety, activity, and immune correlates of anti-PD-1 antibody in cancer.

          Suzanne Topalian, F. Stephen Hodi, Julie Brahmer … (2012)
          Blockade of programmed death 1 (PD-1), an inhibitory receptor expressed by T cells, can overcome immune resistance. We assessed the antitumor activity and safety of BMS-936558, an antibody that specifically blocks PD-1. We enrolled patients with advanced melanoma, non-small-cell lung cancer, castration-resistant prostate cancer, or renal-cell or colorectal cancer to receive anti-PD-1 antibody at a dose of 0.1 to 10.0 mg per kilogram of body weight every 2 weeks. Response was assessed after each 8-week treatment cycle. Patients received up to 12 cycles until disease progression or a complete response occurred. A total of 296 patients received treatment through February 24, 2012. Grade 3 or 4 drug-related adverse events occurred in 14% of patients; there were three deaths from pulmonary toxicity. No maximum tolerated dose was defined. Adverse events consistent with immune-related causes were observed. Among 236 patients in whom response could be evaluated, objective responses (complete or partial responses) were observed in those with non-small-cell lung cancer, melanoma, or renal-cell cancer. Cumulative response rates (all doses) were 18% among patients with non-small-cell lung cancer (14 of 76 patients), 28% among patients with melanoma (26 of 94 patients), and 27% among patients with renal-cell cancer (9 of 33 patients). Responses were durable; 20 of 31 responses lasted 1 year or more in patients with 1 year or more of follow-up. To assess the role of intratumoral PD-1 ligand (PD-L1) expression in the modulation of the PD-1-PD-L1 pathway, immunohistochemical analysis was performed on pretreatment tumor specimens obtained from 42 patients. Of 17 patients with PD-L1-negative tumors, none had an objective response; 9 of 25 patients (36%) with PD-L1-positive tumors had an objective response (P=0.006). Anti-PD-1 antibody produced objective responses in approximately one in four to one in five patients with non-small-cell lung cancer, melanoma, or renal-cell cancer; the adverse-event profile does not appear to preclude its use. Preliminary data suggest a relationship between PD-L1 expression on tumor cells and objective response. (Funded by Bristol-Myers Squibb and others; ClinicalTrials.gov number, NCT00730639.).
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            Initial sequencing and analysis of the human genome.

            E. S. Lander, L M Linton, B Birren … (2001)
            The human genome holds an extraordinary trove of information about human development, physiology, medicine and evolution. Here we report the results of an international collaboration to produce and make freely available a draft sequence of the human genome. We also present an initial analysis of the data, describing some of the insights that can be gleaned from the sequence.
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              RNA-guided human genome engineering via Cas9.

              P. Mali, L. YANG, K. Esvelt … (2013)
              Bacteria and archaea have evolved adaptive immune defenses, termed clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated (Cas) systems, that use short RNA to direct degradation of foreign nucleic acids. Here, we engineer the type II bacterial CRISPR system to function with custom guide RNA (gRNA) in human cells. For the endogenous AAVS1 locus, we obtained targeting rates of 10 to 25% in 293T cells, 13 to 8% in K562 cells, and 2 to 4% in induced pluripotent stem cells. We show that this process relies on CRISPR components; is sequence-specific; and, upon simultaneous introduction of multiple gRNAs, can effect multiplex editing of target loci. We also compute a genome-wide resource of ~190 K unique gRNAs targeting ~40.5% of human exons. Our results establish an RNA-guided editing tool for facile, robust, and multiplexable human genome engineering.
              Article 0 comments Cited 1505 times – based on 0 reviews     Review now
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                Author and article information

                Journal
                Journal ID (nlm-ta): Proc Biol Sci
                Journal ID (iso-abbrev): Proc. Biol. Sci
                Journal ID (publisher-id): RSPB
                Journal ID (hwp): royprsb
                Title: Proceedings of the Royal Society B: Biological Sciences
                Publisher: The Royal Society
                ISSN (Print): 0962-8452
                ISSN (Electronic): 1471-2954
                Publication date (Print): 22 December 2015
                Publication date PMC-release: 22 December 2015
                Volume: 282
                Issue: 1821
                Electronic Location Identifier: 20143003
                Affiliations
                [1 ]Division of Infection and Immunity, University College London , Gower Street, London WC1E 6BT, UK
                [2 ]Institute of Child Health, University College London , Gower Street, London WC1E 6BT, UK
                [3 ]Division of Advanced Therapies, National Institute for Biological Standards and Control , Blanche Lane, Potters Bar, Herts EN6 3QG, UK
                Author notes

                One contribution to the special feature ‘Evolution and genetics in medicine’ Guest edited by Roy Anderson and Brian Spratt.

                Invited to commemorate 350 years of scientific publishing at the Royal Society.

                Article
                Publisher ID: rspb20143003
                DOI: 10.1098/rspb.2014.3003
                PMC ID: 4707739
                PubMed ID: 26702034
                SO-VID: 731cca4e-0930-4269-85e7-083cf99dee12
                Copyright © © 2015 The Authors.
                License:

                © 2015 The Authors. Published by the Royal Society under the terms of the Creative Commons Attribution License http://creativecommons.org/licenses/by/4.0/, which permits unrestricted use, provided the original author and source are credited.

                History
                Date received : 10 December 2014
                Date accepted : 21 April 2015
                Categories
                Subject: 1001
                Subject: 87
                Subject: 197
                Subject: Special Feature
                Subject: Review Article
                Custom metadata
                cover-date December 22, 2015

                ScienceOpen disciplines: Life sciences
                Keywords: gene therapy,evolutionary medicine,personalized medicine
                Data availability:
                ScienceOpen disciplines: Life sciences
                Keywords: gene therapy, evolutionary medicine, personalized medicine

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