We have located links that may give you full text access.
Cardiac gene therapy with adeno-associated virus-based vectors.
Current Opinion in Cardiology 2017 Februrary 5
PURPOSE OF REVIEW: Cardiac gene therapy with adeno-associated virus (AAV)-based vectors is emerging as an entirely new platform to treat, or even cure, so far intractable cardiac disorders. This review describes our current knowledge of cardiac AAV gene therapy with a particular focus on the biggest obstacle for the successful translation of cardiac AAV gene therapy into the clinic, namely the efficient delivery of the therapeutic gene to the myocardium.
RECENT FINDINGS: We summarize the significant recent progress that has been made in treating heart failure in preclinically relevant animal models with AAV gene therapy and the recent results of clinical trials with cardiac AAV gene therapy for the treatment of heart failure. We also discuss the benefits and shortcomings of the currently available delivery methods of AAV to the heart. Finally, we describe the current state of identifying novel AAV variants that have enhanced tropism for human cardiomyocytes and that show increased resistance to preexisting neutralizing antibodies.
SUMMARY: Here, we describe the successes and challenges in cardiac AAV gene therapy, a treatment modality that has the potential to transform current treatment approaches for cardiac diseases.
RECENT FINDINGS: We summarize the significant recent progress that has been made in treating heart failure in preclinically relevant animal models with AAV gene therapy and the recent results of clinical trials with cardiac AAV gene therapy for the treatment of heart failure. We also discuss the benefits and shortcomings of the currently available delivery methods of AAV to the heart. Finally, we describe the current state of identifying novel AAV variants that have enhanced tropism for human cardiomyocytes and that show increased resistance to preexisting neutralizing antibodies.
SUMMARY: Here, we describe the successes and challenges in cardiac AAV gene therapy, a treatment modality that has the potential to transform current treatment approaches for cardiac diseases.
Full text links
Related Resources
Get seemless 1-tap access through your institution/university
For the best experience, use the Read mobile app
All material on this website is protected by copyright, Copyright © 1994-2024 by WebMD LLC.
This website also contains material copyrighted by 3rd parties.
By using this service, you agree to our terms of use and privacy policy.
Your Privacy Choices
You can now claim free CME credits for this literature searchClaim now
Get seemless 1-tap access through your institution/university
For the best experience, use the Read mobile app