After the Nobel, what next for Crispr  gene-editing therapies?

Hailed as the ‘molecular scissors’ that will allow us to rewrite our genes, the DNA tool is being trialled in treatments for everything from sickle-cell anaemia to cancerWhen last year ’s Nobel prize for chemistry was awarded tobiochemist Jennifer Doudna and microbiologist Emmanuelle Charpentier for their work in developing the technique of gene editing known as Crispr-Cas9 (pronounced “crisper”), headlines hailed their discovery as “molecular scissors” that would allow us to “rewrite the book of life” – with all the complicated ethical questions that ability raises. But much of the excitement has nothing to do with visions of designer babies. The real promise of Cri spr is for treating diseases caused by genetic mutations, from muscular dystrophy to congenital blindness, and even some cancers.The first human trials of Crispr therapies are happening already, and researchers hope that they are on the brink of reaching the clinic. “The speed at which Crispr research has progressed has been truly astonishing,” says Doudna from the University of California at Berkeley.Continue reading...
Source: Guardian Unlimited Science - Category: Science Authors: Tags: Gene editing Medical research Genetics Biology Science Cancer Huntington's disease Sickle cell disease Source Type: news

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Source: Journal of Hazardous Materials - Category: Environmental Health Source Type: research
We describe the most highly recommended generic and disease-specific PRO tools in SCD and discuss the challenges of incorporating them in clinical practice. EXPERT OPINION: PRO measures are essential to incorporate into SCD clinical trials either as primary or secondary outcomes. The use of PRO measures in SCD facilitates a patient-centered approach, which is likely to lead to improved outcomes. Significant challenges remain in adapting PRO tools to routine clinical use and in developing countries. PMID: 33034214 [PubMed - as supplied by publisher]
Source: Expert Review of Hematology - Category: Hematology Tags: Expert Rev Hematol Source Type: research
Authors: Musio F Abstract INTRODUCTION: Anemia has and will continue to be a central theme in medicine particularly as clinicians are treating a burgeoning population of complex multi-organ system processes. As a result of multiple randomized controlled trials (RCTs), meta-analyses, and societal recommendations overly restrictive paradigms and under-administration of erythropoiesis stimulating agents (ESAs) have likely been followed by clinicians among all specialties. AREAS COVERED: A review of anemia in the context of chronic kidney disease, hematologic malignancies and cancer is presented with focus on the e...
Source: Expert Review of Hematology - Category: Hematology Tags: Expert Rev Hematol Source Type: research
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Authors: Matti B, Zargar-Shoshtari K Abstract Prostate cancer represents a significant health burden worldwide. The cancer incidence had substantially increased since the introduction of prostate specific antigen (PSA) in cancer screening. This had led to considerable debates among health professionals and epidemiologists, since PSA as a screening tool seemed to be far from perfect. In New Zealand, the controversy was quite prominent in the last three decades, with some advocating the benefits of screening, while others concerned regarding the risk of harms. With the absence of an organised screening programme and ...
Source: New Zealand Medical Journal - Category: General Medicine Tags: N Z Med J Source Type: research
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Authors: Zarrabi A, Mark S PMID: 33032299 [PubMed - in process]
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Source: Expert Opinion on Pharmacotherapy - Category: Drugs & Pharmacology Tags: Expert Opin Pharmacother Source Type: research
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