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

Related Links:

Authors: Heras Benito M, Fernández-Reyes Luis MJ Abstract Chronic kidney disease is common in people>65years of age. The development and improvement of dialysis techniques has allowed its generalisation to the entire population, when there is a situation of terminal nephropathy, without limit of use due to chronological age. Decision making in elderly patients with advanced chronic kidney disease is complex: in addition to renal parameters, both comorbidity and the presence of geriatric syndromes must be considered. This review addresses the management of information, the decision making of different trea...
Source: Medicina Clinica - Category: General Medicine Tags: Med Clin (Barc) Source Type: research
Conclusion: Diabetes was found to be a bigger cause (62.3%) of CKD than what has been reported thus far in India. At presentation association of diabetic-CKD with HT was recorded higher (78.7%) in India. Hence use of the syndrome "DHKD", (complex of diabetes, hypertension and kidney disease) is justifiable. Our study shows 54.4% of diabetic patients attending medicine OPD were uninvestigated by either physician or GP for CKD because urine albumin and serum creatinine tests were lacking. Thus, progression to CKD in many patients went unnoticed. Syndromic diagnosis of "DHKD" therefore in our view is impor...
Source: Journal of the Association of Physicians of India - Category: General Medicine Tags: J Assoc Physicians India Source Type: research
Conclusion: In patients with prosthetic valve replacement, anticoagulant therapy with VKA seems to be an effective option. The role for non-VKA oral anticoagulants in the setting of prosthetic valve replacement has yet to be established. Furthermore, whether the novel oral anticoagulants are safe and efficacious in patients after placement of a bioprosthetic valve remains unanswered. PMID: 30341847 [PubMed - in process]
Source: Journal of the Association of Physicians of India - Category: General Medicine Tags: J Assoc Physicians India Source Type: research
Conclusion: While evaluating syncope most often the battery of investigations does not lead to any conclusive diagnosis. There was male predominance in presentation. Out of 50 patients 10% had cardiogenic, 20% had neurocardiogenic and in 70% diagnosis was not established. Only 20% had HUTT positive. Echocardiography is the investigation of choice in patients of valvular heart disease. A thorough clinical evaluation is must, investigations are of limited value. PMID: 30341843 [PubMed - in process]
Source: Journal of the Association of Physicians of India - Category: General Medicine Tags: J Assoc Physicians India Source Type: research
PURPOSE: The aim of the current study was to evaluate the association between a decreased estimated glomerular filtration rate (eGFR) and accidental falling in elderly patients who visited the day clinic of the department of geriatric medicine of the Unive...
Source: SafetyLit - Category: International Medicine & Public Health Tags: Age: Elder Adults Source Type: news
The incidence of chronic kidney disease (CKD) has reached pandemic proportions across the world. Occlusive arterial disease causing critical limb ischemia and sepsis in the CKD patient is a huge clinical challenge often culminating in major amputation or mortality. Successful revascularization of these patients is challenging with added limitations due to impaired renal function, difficulties in contrast-enhanced imaging, heavily calcified arteries, and overall comorbid status.
Source: Journal of Vascular Surgery - Category: Surgery Authors: Source Type: research
Traditional contrast media are problematic in advanced chronic kidney disease. Ultrasmall superparamagnetic particles of iron oxide (ferumoxytol) are safe in chronic kidney disease and have been used for imaging in other settings. We tested the feasibility of ferumoxytol-enhanced magnetic resonance angiography (FeMRA) in patients with renal failure and performed a dose-finding study.
Source: Journal of Vascular Surgery - Category: Surgery Authors: Source Type: research
We report a specific contrast medium-free approach to patients with abdominal aortic disease and moderate to severe renal insufficiency. The protocol includes accurate assessment of chronic kidney disease (CKD), preoperative ultrasound evaluation, intravascular ultrasound, and AFX2 endoprosthesis (Endologix, Irvine, Calif).
Source: Journal of Vascular Surgery - Category: Surgery Authors: Source Type: research
Radiocephalic arteriovenous fistula (RCAVF) is the “gold standard” vascular access for end-stage chronic kidney disease patients. Exercises after AVF creation improve maturation. No articles regarding neuromuscular electrostimulation (NMES) in AVF maturation have been published.
Source: Journal of Vascular Surgery - Category: Surgery Authors: Source Type: research
AbstractAngiotensin-converting enzyme inhibitors and angiotensin receptor blockers have been the cornerstone for the treatment of heart failure (HF) with reduced ejection fraction for decades. According to recent and promising studies, sacubitril/valsartan (SV) might be efficient in alternative indications in the area of HF with preserved left ventricular ejection fraction, chronic kidney disease, and so on. This review briefly summarizes these promising therapeutic options regarding SV and the potential limits and pitfalls for its use in routine practice (due to cognitive uncertainties).
Source: American Journal of Cardiovascular Drugs - Category: Cardiology Source Type: research
More News: Allergy & Immunology | Babies | Babies Heart Conditions | Boys | Brain | Cardiology | Cardiovascular | Chemistry | Child Development | Children | Chronic Kidney Disease | Databases & Libraries | Gene Therapy | Genetics | Grants | Heart | Heart Disease | Learning | Men | Microbiology | Muscular Dystrophy | Nanotechnology | Neurology | Neuroscience | Reflex Sympathetic Dystrophy | Respiratory Medicine | Science | Universities & Medical Training | Urology & Nephrology | USA Health