New tool for rapidly analyzing CRISPR edits reveals frequent production of unintended edits

(Burness) Amidst rising hopes for using CRISPR gene editing tools to repair deadly mutations linked to conditions like cystic fibrosis and sickle cell disease, a new study in the Nature journal Communications Biology describes a new innovation that could accelerate this work by rapidly revealing unintended and potentially harmful changes introduced by a gene editing process.
Source: EurekAlert! - Biology - Category: Biology Source Type: news

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CONCLUSIONS: We found only one very small randomised controlled trial; this is not enough to make any reliable conclusion to support the use of blood transfusion. Whilst there appears to be some indication that chronic blood transfusion may play a roll in reducing the incidence of acute chest syndrome in people with sickle cell disease and albeit offering transfusions may be a widely accepted clinical practice, there is currently no reliable evidence to support or refute the perceived benefits of these as treatment options; very limited information about any of the potential harms associated with these interventions or ind...
Source: Cochrane Database of Systematic Reviews - Category: General Medicine Authors: Tags: Cochrane Database Syst Rev Source Type: research
CONCLUSIONS: This Cochrane Review did not identify any randomised controlled studies assessing interventions for the treatment of dental complications in people with sickle cell disease. There is an important need for randomised controlled studies in this area, so as to identify the most effective and safe method for treating dental complications in people with sickle cell disease. PMID: 31841224 [PubMed - in process]
Source: Cochrane Database of Systematic Reviews - Category: General Medicine Authors: Tags: Cochrane Database Syst Rev Source Type: research
CONCLUSIONS: We found no evidence that adding hip core decompression to physical therapy achieves clinical improvement in people with sickle cell disease with avascular necrosis of bone compared to physical therapy alone. However, we highlight that our conclusion is based on one trial with high attrition rates. Further randomized controlled trials are necessary to evaluate the role of hip-core depression for this clinical condition. Endpoints should focus on participants' subjective experience (e.g. quality of life and pain) as well as more objective 'time-to-event' measures (e.g. mortality, survival, hip longevity). The a...
Source: Cochrane Database of Systematic Reviews - Category: General Medicine Authors: Tags: Cochrane Database Syst Rev Source Type: research
Plastic bronchitis is a potentially fatal disorder characterized by the exudation of proteinaceous material and cells in the airways that leads to branching cast formation, which can be expectorated or lead to asphyxiation. Plastic bronchitis can appear as a complication after single-ventricle palliation. Other causes of plastic bronchitis that have been described include cystic fibrosis, sickle-cell anemia, asthma, and lymphangiomatosis. The underlying pathophysiology of plastic bronchitis seems to be abnormal pulmonary lymphatic perfusion, and the preferred treatment is embolization of the abnormal pulmonary lymphatic vessels (1).
Source: Journal of Vascular and Interventional Radiology : JVIR - Category: Radiology Authors: Tags: Letter to the Editor Source Type: research
CONCLUSIONS: It is beneficial to give routine malaria chemoprophylaxis in sickle cell disease in areas where malaria is endemic. 4 November 2019 Update pending New contributors needed The CIDG Editors are looking for contributors to update and maintain this Cochrane Review. Contact the CIDG Managing Editor for further information. PMID: 31681984 [PubMed - in process]
Source: Cochrane Database of Systematic Reviews - Category: General Medicine Authors: Tags: Cochrane Database Syst Rev Source Type: research
As of October, 2019 Gideon www.GideonOnline.com and the Gideon e-book series contain details of 69,204 epidemiological surveys – of which 1,107 (1.6%) are related to the prevalence of specific viral species in patients with respiratory tract infection.  [1-3] The following chronology of published studies summarizes the relative proportion of viral agents associated with non-influenza childhood respiratory infection in the United States.  Additional details and primary references are available on request. 1976 – 2001 Tennessee hMPV accounted for 20% of acute respiratory illness among children ages 0 to...
Source: GIDEON blog - Category: Databases & Libraries Authors: Tags: Ebooks Epidemiology ProMED Source Type: blogs
‘Prime editing’ more precise than Crispr-Cas9, but still needs time before use on humansScientists have raised fresh hopes for treating people with genetic disorders by inventing a powerful new molecular tool that, in principle, can correct the vast majority of mutations that cause human genetic diseases.The procedure, named “prime editing”, can mend about 89% of the 75,000 or so harmful mutations known to mangle the human genome and lead to conditions such as cystic fibrosis, sickle cell anaemia, and a nerve-destroying illness called Tay-Sachs disease.Continue reading...
Source: Guardian Unlimited Science - Category: Science Authors: Tags: Gene editing Genetics Biology Science Source Type: news
Authors: Lee L, Smith-Whitley K, Banks S, Puckrein G Abstract Sickle cell disease (SCD) is an inherited blood disorder most common among African American and Hispanic American persons. The disease can cause substantial, long-term, and costly health problems, including infections, stroke, and kidney failure, many of which can reduce life expectancy. Disparities in receiving health care among African Americans and other racial/ethnic minority groups in the United States are well known and directly related to poor outcomes associated with SCD. As an orphan disease-one that affects
Source: Public Health Reports - Category: International Medicine & Public Health Tags: Public Health Rep Source Type: research
CONCLUSIONS: The currently available trials do not provide sufficient evidence to determine the effects (benefits or harms) of using inhaled nitric oxide to treat pain (vaso-occlusive) crises in people with sickle cell disease. Large-scale, long-term trials are needed to provide more robust data in this area. Patient-important outcomes (e.g. measures of pain and time to pain resolution and amounts of analgesics used), as well as use of healthcare services should be measured and reported in a standardized form. PMID: 31603241 [PubMed - as supplied by publisher]
Source: Cochrane Database of Systematic Reviews - Category: General Medicine Authors: Tags: Cochrane Database Syst Rev Source Type: research
CONCLUSIONS: We were unable to identify any relevant trials on the efficacy and safety of the antibiotic treatment approaches for people with sickle cell disease suffering from osteomyelitis. Randomised controlled trials are needed to establish the optimum antibiotic treatment for this condition, however, we do not envisage further trials of this intervention will be conducted, and hence the review will no longer be regularly updated. PMID: 31588556 [PubMed - as supplied by publisher]
Source: Cochrane Database of Systematic Reviews - Category: General Medicine Authors: Tags: Cochrane Database Syst Rev Source Type: research
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