Effective gene therapy of Stargardt disease with PEG-ECO/pGRK1-ABCA4-S/MAR nanoparticles

• Main Authors: Da Sun (Author), Wenyu Sun (Author), Song-Qi Gao (Author), Jonathan Lehrer (Author), Amirreza Naderi (Author), Cheng Wei (Author), Sangjoon Lee (Author), Andrew L. Schilb (Author), Josef Scheidt (Author), Ryan C. Hall (Author), Elias I. Traboulsi (Author), Krzysztof Palczewski (Author), Zheng-Rong Lu (Author)
• Format: Book
• Published: Elsevier, 2022-09-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Stargardt disease (STGD) is the most common form of inherited retinal genetic disorders and is often caused by mutations in ABCA4. Gene therapy has the promise to effectively treat monogenic retinal disorders. However, clinically approved adeno-associated virus (AAV) vectors do not have a loading ca...