How to talk about genome editing and retinal gene therapy

How to talk about genome editing and retinal gene therapy These important subjects have full free online access. The Bulletin is starting a series of reviews centred around gene therapy. The first of these reviews is about presenting the ideas to the public; the second on a specific application; more will follow in future editions. In addition, as usual, the Bulletin has a section to celebrate its archive, (see end of ‘In this Issue’). The first free access review: How to talk about genome editing (pages 5) is by Starr from the Progress Educational Trust, London. He says that human genome editing is an area of growing prominence, with many potential therapeutic applications and describes a project by two UK charities, whose participants included fertility sector patients and practitioners and people affected by genetic disease and rare disease. The way genome editing is discussed is often inconsistent and confusing. People’s understanding should ideally be able to withstand and evolve alongside current developments in genome editing, rather than being tied firmly to specific aspects of genome editing. The second chosen free-to-view review is entitled: Retinal Gene Therapy (Pages 13) by Bainbridge from the Institute of Ophthalmology, London. He says that inherited retinal diseases are the leading cause of sight impairment in people of working age in England and Wales, and the second commonest in childhood. Gene therapy offers the potential for benefit. The magnitude of vision improvement from gene therapy is suboptimal, and its durability may be limited by progressive retinal degeneration. The safety and potential benefit of gene therapy for inherited and acquired retinal diseases is being explored in a rapidly expanding number of trials. In the rest of the Bulletin, the third review, also about genetics is entitled Genetic counselling in the era of genomic medicine (pages 27) by Patch and Middleton from King’s College and Queen Mary University, London and the Wellcome Genome Campus and University of Cambridge, UK. They say that genomic technology can now deliver cost-effective, targeted diagnosis and treatment for patients. Genetic counselling is a communication process empowering patients and families to make autonomous decisions and effectively use new genetic information. An international consensus on the definition of genetic counselling has been agreed. Genetic counselling is necessary for the implementation of genomic medicine, but models of genetic counselling are still controversial. The fourth review is entitled: Fortune and hindsight: gene patents’ muted effect on medical practice (Pages 37) by Sherkov and Abbott from New York Law School, USA and the University of Surrey, UK. They say that physicians have long worried about gene patents’ potential to restrict their medical practices. Traditional gene patents do not appear to have restricted medical practice in the UK, although their effect elsewhere has been more nuanced. Continuing survey data on practitioners’ views concerning patents’ role in the distribution of newer technologies would be beneficial. The fifth review is entitled: Conscientious Objection in Medicine: Accommodation versus Professionalism and the Public Good (pages 47) by Schuklenk from Queen’s University, Ontario, Canada. He says that, in recent years, questions have arisen about the moral justification for the accommodation of health care professionals who refuse, on conscience grounds as opposed to professional grounds, to provide services to eligible patients. Central to concerns about conscientious objectors is that health care professionals volunteer to join their professions, that, typically, they are the monopoly providers of such services and that a health care professional’s refusal to provide professional services on grounds that are not professional judgments amount to unprofessional conduct. The sixth review is entitled: Maternal Vitamin D Supplementation during Pregnancy (Pages 57) by Curtis, Moon, Harvey and Cooper from the University of Southampton, UK. They say that Maternal Vitamin D status in pregnancy has been linked to many health outcomes in mother and offspring. The findings are generally inconsistent in most areas, and, at present, there is a lack of data from high-quality intervention studies to confirm a causal role for vitamin D in these outcomes. In most areas, the evidence tends towards maternal vitamin D being of overall benefit, but often does not reach statistical significance in meta-analyses. The seventh review is: Dubious space for Artelon joint resurfacing for basal thumb (trapeziometacarpal joint) osteoarthritis (Pages 79) by Smeraglia, Mariconda, Balato, Di Donato, Criscuolo and Maffulli from the University of Naples, University of Salerno, Italy and the Barts and London School of Medicine and Dentristry, UK. They say that trapeziometacarpal arthritis is a common and disabling condition. There is no evidence in the literature of superiority of one surgical procedure over others. Several prosthetic implants have been introduced to preserve joint mobility. The use of Artelon implant is not recommended because of its high revision rate and worse outcomes compared to conventional techniques. Standard techniques such as trapeziectomy alone or combined with interposition or suspensionplasty offer effective treatment for thumb basal joint arthritis. The eighth review is entitled The cost-effectiveness of follow-up strategies after cancer treatment (Pages 85) by Barbieri, Richardson and Paisley from the University of York and the University of Sheffield, UK. They say that the cost of treatment and follow-up of cancer patients in the UK is substantial. In a budget-constrained system such as the NHS, it is necessary to consider the cost-effectiveness of the range of management strategies at different points on cancer patients’ care pathways to ensure that they provide adequate value for money. Interventions and strategies for follow-up in cancer patients were variable across type of cancer and setting. Drawing general conclusions about the cost-effectiveness of these interventions/strategies is difficult. Where there was evidence, in the most part, NICE guidance broadly reflected this evidence. The ninth review is entitled: CD36 and Lipid Metabolism in the Evolution of Atherosclerosis, (Pages 101) by Zhao, Varghese, Moorhead, Chen and Ruan from Chongqing Medical University and Zhejiang Universit, Hangzhou, China and University College, London. They state that CD36 is a multifunctional class B scavenger receptor, which acts as an important modulator of lipid homoeostasis and immune responses. Abnormally up-regulated CD36 promotes inflammation, foam cell formation, endothelial apoptosis, macrophage trapping and thrombosis. The modulation of CD36 function at post-translational levels has recently been suggested to be a potential therapeutic strategy. The 10th review is entitled: Common mental disorders and work (pages 113) by Nicholson, an Independent Corporate Medical Expert, London. He states that government and employers’ organizations have a sustained focus on common mental disorders, how they impact a person’s ability to gain or continue work, the costs of sickness absence, presenteeism and job loss and the positive impact of work on wellbeing. Common mental disorders are a major cause of disability, unemployment and dependency on welfare benefits. There is growing interest in, and an increasing number of, best practice guidelines for employers, that could be improved and shared more actively. There is need for high-quality studies of interventions that examine work outcomes in patients who have been unable to obtain or continue work because of common mental disorders The 11th review is entitled: Treatment of type 2 diabetes: future approaches (Pages 123) by Bailey and Day from Aston University, UK They say that type 2 diabetes, which accounts for about 90% of all diabetes, is a heterogeneous and progressive disease with a variety of causative and potentiating factors. The hyperglycaemia of type 2 diabetes is often inadequately controlled, hence the need for a wider selection of glucose-lowering treatments. Early, effective and sustained control of blood glucose defers the onset and reduces the severity of microvascular and neuropathic complications of type 2 diabetes and helps to reduce the risk of cardiovascular complications. Newer glucose-lowering agents require extensive long-term studies to confirm cardiovascular safety. From the archive The British Medical Bulletin (BMB) has an extensive archive dating back to 1943. The journal is publishing selected papers from that archive. This quarter we have a piece, the front page of which can be accessed directly online, and the rest read in the link below. This quarter I have chosen a piece by Magnus Pyke; Some principles of war-time food policy, written in 1943 when the war was at its height. Magnus Pyke was nutritional scientist with the Ministry of Food during the war and a well-known scientific personality on radio and TV after the War. There are some fascinating insights into why fruit was so scarce during rationing and why free milk for children was considered so important. The original link is: https://academic.oup.com/bmb/article/2/10-11/228/322076. You can access these collected articles more easily and more fully by visiting the ‘Highlights from the BMB Archive’ collection: (http://bit.ly/2nTsFIH)’. Enjoy! NJV April 2018 © The Author(s) 2018. Published by Oxford University Press. All rights reserved. For permissions, please e-mail: journals.permissions@oup.com This article is published and distributed under the terms of the Oxford University Press, Standard Journals Publication Model (https://academic.oup.com/journals/pages/about_us/legal/notices) http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png British Medical Bulletin Oxford University Press

How to talk about genome editing and retinal gene therapy

British Medical Bulletin , Volume Advance Article (1) – May 17, 2018

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Abstract

These important subjects have full free online access. The Bulletin is starting a series of reviews centred around gene therapy. The first of these reviews is about presenting the ideas to the public; the second on a specific application; more will follow in future editions. In addition, as usual, the Bulletin has a section to celebrate its archive, (see end of ‘In this Issue’). The first free access review: How to talk about genome editing (pages 5) is by Starr from the Progress Educational Trust, London. He says that human genome editing is an area of growing prominence, with many potential therapeutic applications and describes a project by two UK charities, whose participants included fertility sector patients and practitioners and people affected by genetic disease and rare disease. The way genome editing is discussed is often inconsistent and confusing. People’s understanding should ideally be able to withstand and evolve alongside current developments in genome editing, rather than being tied firmly to specific aspects of genome editing. The second chosen free-to-view review is entitled: Retinal Gene Therapy (Pages 13) by Bainbridge from the Institute of Ophthalmology, London. He says that inherited retinal diseases are the leading cause of sight impairment in people of working age in England and Wales, and the second commonest in childhood. Gene therapy offers the potential for benefit. The magnitude of vision improvement from gene therapy is suboptimal, and its durability may be limited by progressive retinal degeneration. The safety and potential benefit of gene therapy for inherited and acquired retinal diseases is being explored in a rapidly expanding number of trials. In the rest of the Bulletin, the third review, also about genetics is entitled Genetic counselling in the era of genomic medicine (pages 27) by Patch and Middleton from King’s College and Queen Mary University, London and the Wellcome Genome Campus and University of Cambridge, UK. They say that genomic technology can now deliver cost-effective, targeted diagnosis and treatment for patients. Genetic counselling is a communication process empowering patients and families to make autonomous decisions and effectively use new genetic information. An international consensus on the definition of genetic counselling has been agreed. Genetic counselling is necessary for the implementation of genomic medicine, but models of genetic counselling are still controversial. The fourth review is entitled: Fortune and hindsight: gene patents’ muted effect on medical practice (Pages 37) by Sherkov and Abbott from New York Law School, USA and the University of Surrey, UK. They say that physicians have long worried about gene patents’ potential to restrict their medical practices. Traditional gene patents do not appear to have restricted medical practice in the UK, although their effect elsewhere has been more nuanced. Continuing survey data on practitioners’ views concerning patents’ role in the distribution of newer technologies would be beneficial. The fifth review is entitled: Conscientious Objection in Medicine: Accommodation versus Professionalism and the Public Good (pages 47) by Schuklenk from Queen’s University, Ontario, Canada. He says that, in recent years, questions have arisen about the moral justification for the accommodation of health care professionals who refuse, on conscience grounds as opposed to professional grounds, to provide services to eligible patients. Central to concerns about conscientious objectors is that health care professionals volunteer to join their professions, that, typically, they are the monopoly providers of such services and that a health care professional’s refusal to provide professional services on grounds that are not professional judgments amount to unprofessional conduct. The sixth review is entitled: Maternal Vitamin D Supplementation during Pregnancy (Pages 57) by Curtis, Moon, Harvey and Cooper from the University of Southampton, UK. They say that Maternal Vitamin D status in pregnancy has been linked to many health outcomes in mother and offspring. The findings are generally inconsistent in most areas, and, at present, there is a lack of data from high-quality intervention studies to confirm a causal role for vitamin D in these outcomes. In most areas, the evidence tends towards maternal vitamin D being of overall benefit, but often does not reach statistical significance in meta-analyses. The seventh review is: Dubious space for Artelon joint resurfacing for basal thumb (trapeziometacarpal joint) osteoarthritis (Pages 79) by Smeraglia, Mariconda, Balato, Di Donato, Criscuolo and Maffulli from the University of Naples, University of Salerno, Italy and the Barts and London School of Medicine and Dentristry, UK. They say that trapeziometacarpal arthritis is a common and disabling condition. There is no evidence in the literature of superiority of one surgical procedure over others. Several prosthetic implants have been introduced to preserve joint mobility. The use of Artelon implant is not recommended because of its high revision rate and worse outcomes compared to conventional techniques. Standard techniques such as trapeziectomy alone or combined with interposition or suspensionplasty offer effective treatment for thumb basal joint arthritis. The eighth review is entitled The cost-effectiveness of follow-up strategies after cancer treatment (Pages 85) by Barbieri, Richardson and Paisley from the University of York and the University of Sheffield, UK. They say that the cost of treatment and follow-up of cancer patients in the UK is substantial. In a budget-constrained system such as the NHS, it is necessary to consider the cost-effectiveness of the range of management strategies at different points on cancer patients’ care pathways to ensure that they provide adequate value for money. Interventions and strategies for follow-up in cancer patients were variable across type of cancer and setting. Drawing general conclusions about the cost-effectiveness of these interventions/strategies is difficult. Where there was evidence, in the most part, NICE guidance broadly reflected this evidence. The ninth review is entitled: CD36 and Lipid Metabolism in the Evolution of Atherosclerosis, (Pages 101) by Zhao, Varghese, Moorhead, Chen and Ruan from Chongqing Medical University and Zhejiang Universit, Hangzhou, China and University College, London. They state that CD36 is a multifunctional class B scavenger receptor, which acts as an important modulator of lipid homoeostasis and immune responses. Abnormally up-regulated CD36 promotes inflammation, foam cell formation, endothelial apoptosis, macrophage trapping and thrombosis. The modulation of CD36 function at post-translational levels has recently been suggested to be a potential therapeutic strategy. The 10th review is entitled: Common mental disorders and work (pages 113) by Nicholson, an Independent Corporate Medical Expert, London. He states that government and employers’ organizations have a sustained focus on common mental disorders, how they impact a person’s ability to gain or continue work, the costs of sickness absence, presenteeism and job loss and the positive impact of work on wellbeing. Common mental disorders are a major cause of disability, unemployment and dependency on welfare benefits. There is growing interest in, and an increasing number of, best practice guidelines for employers, that could be improved and shared more actively. There is need for high-quality studies of interventions that examine work outcomes in patients who have been unable to obtain or continue work because of common mental disorders The 11th review is entitled: Treatment of type 2 diabetes: future approaches (Pages 123) by Bailey and Day from Aston University, UK They say that type 2 diabetes, which accounts for about 90% of all diabetes, is a heterogeneous and progressive disease with a variety of causative and potentiating factors. The hyperglycaemia of type 2 diabetes is often inadequately controlled, hence the need for a wider selection of glucose-lowering treatments. Early, effective and sustained control of blood glucose defers the onset and reduces the severity of microvascular and neuropathic complications of type 2 diabetes and helps to reduce the risk of cardiovascular complications. Newer glucose-lowering agents require extensive long-term studies to confirm cardiovascular safety. From the archive The British Medical Bulletin (BMB) has an extensive archive dating back to 1943. The journal is publishing selected papers from that archive. This quarter we have a piece, the front page of which can be accessed directly online, and the rest read in the link below. This quarter I have chosen a piece by Magnus Pyke; Some principles of war-time food policy, written in 1943 when the war was at its height. Magnus Pyke was nutritional scientist with the Ministry of Food during the war and a well-known scientific personality on radio and TV after the War. There are some fascinating insights into why fruit was so scarce during rationing and why free milk for children was considered so important. The original link is: https://academic.oup.com/bmb/article/2/10-11/228/322076. You can access these collected articles more easily and more fully by visiting the ‘Highlights from the BMB Archive’ collection: (http://bit.ly/2nTsFIH)’. Enjoy! NJV April 2018 © The Author(s) 2018. Published by Oxford University Press. All rights reserved. For permissions, please e-mail: journals.permissions@oup.com This article is published and distributed under the terms of the Oxford University Press, Standard Journals Publication Model (https://academic.oup.com/journals/pages/about_us/legal/notices)

Journal

British Medical BulletinOxford University Press

Published: May 17, 2018

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