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...
CONCLUSIONS: D-FP21-PEG-PEI/plasmid DNA is a safe and efficient gene delivery vehicle for ovarian cancer targeted therapy. PMID: 29667478 [PubMed - in process]
Authors: Rafael D, Gener P, Andrade F, Seras-Franzoso J, Montero S, Fernández Y, Hidalgo M, Arango D, Sayós J, Florindo HF, Abasolo I, Schwartz S, Videira M Abstract Development of RNA interference-based therapies with appropriate therapeutic window remains a challenge for advanced cancers. Because cancer stem cells (CSC) are responsible of sustaining the metastatic spread of the disease to distal organs and the progressive gain of resistance of advanced cancers, new anticancer therapies should be validated specifically for this subpopulation of cells. A new amphihilic-based gene delivery system that ...
CONCLUSIONS: Patients with high hyperopia often have ocular comorbidities. Such eyes may be surgically challenging, resulting in reduced benefits from cataract surgery compared to normal eyes. PMID: 29669369 [PubMed - in process]
CONCLUSIONS: This study shows that anterior lamellar keratoplasty in children retains good visual function when combined with adequate amblyopic therapy. However, the rate of complications is higher in juveniles and requires more intensive interdisciplinary follow-up. PMID: 29669367 [PubMed - in process]
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Funding Opportunity RFA-AI-18-016 from the NIH Guide for Grants and Contracts. This Funding Opportunity Announcement (FOA) will support research on the development and validation of innovative strategies to deliver anti-HIV gene therapies efficiently to specific target cells in vivo.
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Conditions: Keratoconus; Corneal Opacity; Corneal Dystrophy Intervention: Device: Optical Coherence Tomography Sponsor: Oregon Health and Science University Not yet recruiting
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