Wu, Zhijian

Effect of genome size on AAV vector packaging

Wu, Z., Yang, H., & Colosi, P. (2010) · Molecular Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 1,029

Nrl knockdown by AAV-delivered CRISPR/Cas9 prevents retinal degeneration in mice

Yu, W., Mookherjee, S., Chaitankar, V., Hiriyanna, S., Kim, J.-W., Brooks, M., Ataeijannati, Y., Sun, X., Dong, L., Li, T., et al. (2017) · Nature Communications

Retinal Development and DisordersCRISPR and Genetic Engineering

Cited by 271

Retinal AAV8-RS1 Gene Therapy for X-Linked Retinoschisis: Initial Findings from a Phase I/IIa Trial by Intravitreal Delivery

Cukras, C., Wiley, H. E., Jeffrey, B. G., Sen, H. N., Turriff, A., Zeng, Y., Vijayasarathy, C., Marangoni, D., Ziccardi, L., Kjellstrom, S., et al. (2018) · Molecular Therapy

Retinal Development and DisordersVirus-based gene therapy research

Cited by 268

Engineering and Selection of Shuffled AAV Genomes: A New Strategy for Producing Targeted Biological Nanoparticles

Li, W., Asokan, A., Wu, Z., Van Dyke, T., DiPrimio, N., Johnson, J. S., Govindaswamy, L., Agbandje-McKenna, M., Leichtle, S., Eugene Redmond Jr, D., et al. (2008) · Molecular Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 261

Optimization of self-complementary AAV vectors for liver-directed expression results in sustained correction of hemophilia B at low vector dose

Wu, Z., Sun, J., Zhang, T., Yin, C., Yin, F., Van Dyke, T., Samulski, R. J., & Monahan, P. E. (2008) · Molecular Therapy

Virus-based gene therapy researchHemophilia Treatment and Research

Cited by 153

Employing a Gain-of-Function Factor IX Variant R338L to Advance the Efficacy and Safety of Hemophilia B Human Gene Therapy: Preclinical Evaluation Supporting an Ongoing Adeno-Associated Virus Clinical Trial

Monahan, P. E., Sun, J., Gui, T., Hu, G., Hannah, W. B., Wichlan, D. G., Wu, Z., Grieger, J. C., Li, C., Suwanmanee, T., et al. (2015) · Human Gene Therapy

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 108

Preclinical dose-escalation study of intravitreal AAV-RS1 gene therapy in a mouse model of X-linked retinoschisis: dose-dependent expression and improved retinal structure and function

Bush, R. A., Zeng, Y., Colosi, P., Kjellstrom, S., Hiriyanna, S., Vijayasarathy, C., Santos, M., Li, J., Wu, Z., & Sieving, P. A. (2016) · Human Gene Therapy

Retinal Development and DisordersVirus-based gene therapy research

Cited by 86

A CEP290 C-Terminal Domain Complements the Mutant CEP290 of Rd16 Mice In Trans and Rescues Retinal Degeneration

Mookherjee, S., Chen, H. Y., Isgrig, K., Yu, W., Hiriyanna, S., Levron, R., Li, T., Colosi, P., Chien, W., Swaroop, A., et al. (2018) · Cell Reports

Retinal Development and DisordersGenetic and Kidney Cyst Diseases

Cited by 41

Trans-ocular Electric Current In Vivo Enhances AAV-Mediated Retinal Gene Transduction after Intravitreal Vector Administration

Song, H., Bush, R. A., Zeng, Y., Qian, H., Wu, Z., & Sieving, P. A. (2019) · Molecular Therapy - Methods & Clinical Development

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 17

AAVrh-10 transduces outer retinal cells in rodents and rabbits following intravitreal administration

Zeng, Y., Qian, H., Wu, Z., Marangoni, D., Sieving, P. A., & Bush, R. A. (2019) · Gene Therapy

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 12

Trans-Ocular Electric Current In Vivo Enhances AAV-Mediated Retinal Transduction in Large Animal Eye After Intravitreal Vector Administration

Song, H., Zeng, Y., Sardar Pasha, S. P. B., Bush, R. A., Vijayasarathy, C., Qian, H., Wei, L., Wiley, H. E., Wu, Z., & Sieving, P. A. (2020) · Translational Vision Science & Technology

Retinal Development and DisordersVirus-based gene therapy research

Cited by 9