UCLA research could be first step toward healing the hearts of children with Duchenne

After a progressive weakening of the muscles takes away their motor skills, and then their abilities to stand and walk, most males with Duchenne muscular dystrophy die of heart and respiratory failure in their 20s.Now, researchers at theDavid Geffen School of Medicine at UCLA are pursuing a cutting-edge way to stop heart disease in patients with Duchenne, which affects one in 5,000 male babies born in the United States. Their work, which is supported by a David Geffen School of Medicine Seed Grant, is just one of a number of projects underway at the medical school in which interdisciplinary groups of UCLA researchers are pursuing new treatments and cures.The scientists are trying to use minuscule nanoparticles to carry bits of DNA to the hearts of people with Duchenne. Inside heart cells, this new DNA would “trick” the cells’ machinery to reverse the genetic defect. The team is made up of Huan Meng, an assistant professor of nanomedicine; April Pyle, an associate professor of microbiology, immunology and molecular genetics; and Melissa Spencer, a professor of neurology.“Duchenne is such a complicated genetic disease,” Spencer said. “However, because we know the defective gene, recent advances let us directly edit the abnormal gene in muscle and heart cells in vivo.”The complex work is possible because it draws on the researchers ’ varied areas of expertise: Meng’s experience designing drug delivery systems using the latest...
Source: UCLA Newsroom: Health Sciences - Category: Universities & Medical Training Source Type: news

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The American Society of Clinical Oncology/College of American Pathologists (ASCO/CAP) 2013 guidelines for HER2 assessment have increased the number of HER2 equivocal breast carcinomas following in situ hybridization reflex testing, that is, HER2 “double equivocal” (equivocal protein expression and equivocal gene copy number). Forty-five double-equivocal carcinomas were subjected to Prosigna analysis. Twenty-seven cases were investigated for the expression of genes found to be differentially expressed between estrogen receptor (ER)-positive/HER2-positive (N=22) and ER-positive/HER2-negative (N=22) control cases....
Source: The American Journal of Surgical Pathology - Category: Pathology Tags: Original Articles Source Type: research
Objectives: To investigate the risk of stroke in SSNHL patients. Study Design: A longitudinal follow-up cohort study. Setting: The Korean National Health Insurance Service-National Sample Cohort from 2002 to 2013. Patients: The 4,944 SSNHL participants were matched with 19,776 controls for age, sex, income, region of residence, hypertension, diabetes, and dyslipidemia. The SSNHL subjects were diagnosed (International Classification of Disease [ICD]-10: H91.2), underwent an audiometry examination, and were treated with steroids. The history of hemorrhagic stroke, ischemic stroke, hypertension, diabetes, dyslipide...
Source: Otology and Neurotology - Category: ENT & OMF Tags: Sens Orineural Hearing Loss and Tinnitus Source Type: research
Conclusion: Bilateral PSCC occlusion results in a reduction in the vertical VOR of the PSCCs. Over a 6 to 12 month time period improvement in the VOR gain can be demonstrated, most likely due to central compensation. There is clinical correlation with improvement in DVA testing. Bilateral PSCC occlusion is a safe and effective treatment for bilateral BPV proved refractory to particle repositioning manoeuvers.
Source: Otology and Neurotology - Category: ENT & OMF Tags: Vestibular Disorders Source Type: research
A morbidly-obese 57-year-old diabetic and hypertensive man with chronic kidney disease, diastolic heart failure, and bilateral hearing loss was found to have soft tissue masses/densities in the lateral aspect of both auditory canals on local examination and on imaging. He underwent biopsies of both ear canal masses and histologic examination revealed amyloid deposits in the dermis. These deposits were confirmed as AL (amyloid light chain) kappa-type amyloid by laser mass spectrometry. A systemic work-up showed plasma cell dyscrasia with 9% kappa light chain restricted plasma cells in the bone marrow as well as amyloid depo...
Source: Otology and Neurotology - Category: ENT & OMF Tags: Temporal Bone Histopathology Case of the Month Source Type: research
DiscussionAdherence to treatment recommendations is crucial for the successful management of FH and subsequent decrease in risk of CHD later in life. Common identified themes could provide an understanding of the beliefs and experiences which influence adherence to treatment recommendations and provide an insight into perceived barriers and facilitators. The findings are intended to be used in the development of future interventions or guidelines regarding treatment of children and adults with FH.Systematic review registrationPROSPERO registration number:CRD42018085946
Source: Systematic Reviews - Category: International Medicine & Public Health Source Type: research
ConclusionsIn a group of hemodialysis patients from one centre, T-score in proximal femur, low vitamin D, low BMI, and high FRAX for major osteoporotic fracture were associated with low-trauma fractures, however, in multi-variate analysis only low BMI remained a significant predictor of fracture risk.
Source: International Urology and Nephrology - Category: Urology & Nephrology Source Type: research
Authors: Charytoniuk T, Małyszko M, Bączek J, Fiedorczyk P, Siedlaczek K, Małyszko J Abstract Nephrectomy, which constitutes a gold-standard procedure for the treatment of renal-cell carcinoma (RCC), has been widely discussed in the past decade as a significant risk factor of the development of chronic kidney disease (CKD). RCC is the third most common genitourinary cancer in the United States, with an estimated more than 65,000 new cases and 14,970 deaths. The aim of this review was to precisely and comprehensively summarize the status of current knowledge in chronic kidney disease risk factors after nephrectom...
Source: Postgraduate Medicine - Category: Internal Medicine Tags: Postgrad Med Source Type: research
U.S. health officials revised rules for gene therapy experiments
Source: ABC News: Health - Category: Consumer Health News Tags: Health Source Type: news
U.S. health officials revised rules for gene therapy experiments
Source: ABC News: Health - Category: Consumer Health News Tags: Health Source Type: news
A growing number of studies suggest that getting a handle on a few key risk factors can bring type 2 diabetes under control. Lowering blood sugar, for instance, reduces the risk of additional health problems, such as heart disease and stroke related to the disease. But most of these studies have focused on studying one risk factor — like blood sugar, cholesterol or blood pressure — at a time. In a new study published in the New England Journal of Medicine, researchers analyzed what happens to death rates and other health problems when people control up to five known risk factors for type 2 diabetes. The study ...
Source: TIME: Health - Category: Consumer Health News Authors: Tags: Uncategorized healthytime medicine Source Type: news
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