WebDec 1, 2024 · The authors describe several emerging cardiomyopathy treatments, including gene therapy, direct targeting of myofilament function, protein quality control, metabolism, and others. The authors discuss advantages and disadvantages of these approaches and indicate areas of high potential for short- and longer term efficacy. WebMar 31, 2024 · The purpose of this study is to test the safety and preliminary efficacy of AAVrh.10hFXN to treat the cardiomyopathy associated with Friedreich's ataxia (FA). AAVrh.10hFXN is a serotype rh.10 adeno-associated virus gene transfer vector coding for Frataxin (FXN). The drug is administered intravenously.
CRISPR gene-editing therapies for hypertrophic …
WebApr 1, 2024 · Evaluation of gene expression in the pediatric patients is currently underway. Conclusions: Robust immunomodulation during the initial days post-infusion enabled administration of this first-in-pediatric RP-A501 gene therapy for DD cardiomyopathy without evidence of immune-medi- ated clinical sequelae. WebJan 24, 2024 · Only a few companies are using gene therapy to treat heart disease. One of them, Tenaya Therapeutics, is using adeno-associated virus 9 (AAV9) to deliver healthy copies of select genes to target cardiac tissue and express the protein of interest at high levels.What’s particularly interesting is that it also has a cell regeneration program that – … chandigarh to mumbai flight tickets
Lamin A/C Cardiomyopathy: Implications for Treatment
WebMar 13, 2024 · Heart disease remains the prevalent cause of premature death and accounts for a significant proportion of all hospital admissions. Molecular genetics was integrated … WebJul 21, 2024 · Through the agreement, LEXEO obtains exclusive rights to three preclinical AAV-mediated gene therapy programs focused on TNNI3-associated hypertrophic cardiomyopathy (HCM) and arrhythmogenic right ... WebMar 2, 2024 · This indicates that the therapy designed and applied does not have to be perfect (100% correction) to be effective. In addition, LMNA is a well-known gene in cardiovascular disease and is linked to dilated cardiomyopathy and arrhythmias. At the same time, Duchenne patients often develop dilated cardiomyopathy. chandigarh to nagpur flight