Unexpected mutations after CRISPR–Cas9 editing in vivo
Schaefer, Kellie A, Wu, Wen-Hsuan, Colgan, Diana F, Tsang, Stephen H, Bassuk, Alexander G, Mahajan, Vinit B
Published in Nature methods (30.05.2017)
Published in Nature methods (30.05.2017)
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Allele-Specific Chromosome Removal after Cas9 Cleavage in Human Embryos
Zuccaro, Michael V., Xu, Jia, Mitchell, Carl, Marin, Diego, Zimmerman, Raymond, Rana, Bhavini, Weinstein, Everett, King, Rebeca T., Palmerola, Katherine L., Smith, Morgan E., Tsang, Stephen H., Goland, Robin, Jasin, Maria, Lobo, Rogerio, Treff, Nathan, Egli, Dieter
Published in Cell (10.12.2020)
Published in Cell (10.12.2020)
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Precision Medicine: Genetic Repair of Retinitis Pigmentosa in Patient-Derived Stem Cells
Bassuk, Alexander G., Zheng, Andrew, Li, Yao, Tsang, Stephen H., Mahajan, Vinit B.
Published in Scientific reports (27.01.2016)
Published in Scientific reports (27.01.2016)
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Clustered Regularly Interspaced Short Palindromic Repeats-Based Genome Surgery for the Treatment of Autosomal Dominant Retinitis Pigmentosa
Tsai, Yi-Ting, Wu, Wen-Hsuan, Lee, Ting-Ting, Wu, Wei-Pu, Xu, Christine L., Park, Karen S., Cui, Xuan, Justus, Sally, Lin, Chyuan-Sheng, Jauregui, Ruben, Su, Pei-Yin, Tsang, Stephen H.
Published in Ophthalmology (Rochester, Minn.) (01.09.2018)
Published in Ophthalmology (Rochester, Minn.) (01.09.2018)
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Adeno-Associated Viral Gene Therapy for Inherited Retinal Disease
Ong, Tuyen, Pennesi, Mark E., Birch, David G., Lam, Byron L., Tsang, Stephen H.
Published in Pharmaceutical research (01.02.2019)
Published in Pharmaceutical research (01.02.2019)
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Rare and common variants in ROM1 and PRPH2 genes trans-modify Stargardt/ABCA4 disease
Zernant, Jana, Lee, Winston, Wang, Jun, Goetz, Kerry, Ullah, Ehsan, Nagasaki, Takayuki, Su, Pei-Yin, Fishman, Gerald A, Tsang, Stephen H, Tumminia, Santa J, Brooks, Brian P, Hufnagel, Robert B, Chen, Rui, Allikmets, Rando
Published in PLoS genetics (30.03.2022)
Published in PLoS genetics (30.03.2022)
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Mutations in the unfolded protein response regulator ATF6 cause the cone dysfunction disorder achromatopsia
Kohl, Susanne, Zobor, Ditta, Chiang, Wei-Chieh, Weisschuh, Nicole, Staller, Jennifer, Menendez, Irene Gonzalez, Chang, Stanley, Beck, Susanne C, Garrido, Marina Garcia, Sothilingam, Vithiyanjali, Seeliger, Mathias W, Stanzial, Franco, Benedicenti, Francesco, Inzana, Francesca, Héon, Elise, Vincent, Ajoy, Beis, Jill, Strom, Tim M, Rudolph, Günther, Roosing, Susanne, Hollander, Anneke I den, Cremers, Frans P M, Lopez, Irma, Ren, Huanan, Moore, Anthony T, Webster, Andrew R, Michaelides, Michel, Koenekoop, Robert K, Zrenner, Eberhart, Kaufman, Randal J, Tsang, Stephen H, Wissinger, Bernd, Lin, Jonathan H
Published in Nature genetics (01.07.2015)
Published in Nature genetics (01.07.2015)
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Journal Article
Primary versus Secondary Elevations in Fundus Autofluorescence
Parmann, Rait, Tsang, Stephen H, Sparrow, Janet R
Published in International journal of molecular sciences (01.08.2023)
Published in International journal of molecular sciences (01.08.2023)
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Quantitative progression of retinitis pigmentosa by optical coherence tomography angiography
Jauregui, Ruben, Park, Karen Sophia, Duong, Jimmy K., Mahajan, Vinit B., Tsang, Stephen H.
Published in Scientific reports (03.09.2018)
Published in Scientific reports (03.09.2018)
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Journal Article
CRISPR Repair Reveals Causative Mutation in a Preclinical Model of Retinitis Pigmentosa
Wu, Wen-Hsuan, Tsai, Yi-Ting, Justus, Sally, Lee, Ting-Ting, Zhang, Lijuan, Lin, Chyuan-Sheng, Bassuk, Alexander G, Mahajan, Vinit B, Tsang, Stephen H
Published in Molecular therapy (01.08.2016)
Published in Molecular therapy (01.08.2016)
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Journal Article
HTRA1, an age‐related macular degeneration protease, processes extracellular matrix proteins EFEMP1 and TSP1
Lin, Michael K., Yang, Jin, Hsu, Chun Wei, Gore, Anuradha, Bassuk, Alexander G., Brown, Lewis M., Colligan, Ryan, Sengillo, Jesse D., Mahajan, Vinit B., Tsang, Stephen H.
Published in Aging cell (01.08.2018)
Published in Aging cell (01.08.2018)
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Journal Article
CRISPR/Cas therapeutic strategies for autosomal dominant disorders
Caruso, Salvatore Marco, Quinn, Peter Mj, da Costa, Bruna Lopes, Tsang, Stephen H
Published in The Journal of clinical investigation (01.05.2022)
Published in The Journal of clinical investigation (01.05.2022)
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CRISPR editing demonstrates rs10490924 raised oxidative stress in iPSC-derived retinal cells from patients with ARMS2/HTRA1 -related AMD
Chang, Ya-Ju, Jenny, Laura A, Li, Yong-Shi, Cui, Xuan, Kong, Yang, Li, Yao, Sparrow, Janet R, Tsang, Stephen H
Published in Proceedings of the National Academy of Sciences - PNAS (09.05.2023)
Published in Proceedings of the National Academy of Sciences - PNAS (09.05.2023)
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Journal Article
Quantitative Fundus Autofluorescence Distinguishes ABCA4 -Associated and Non– ABCA4 -Associated Bull's-Eye Maculopathy
Duncker, Tobias, MD, Tsang, Stephen H., MD, PhD, Lee, Winston, MA, Zernant, Jana, MS, Allikmets, Rando, PhD, Delori, François C., PhD, Sparrow, Janet R., PhD
Published in Ophthalmology (Rochester, Minn.) (01.02.2015)
Published in Ophthalmology (Rochester, Minn.) (01.02.2015)
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Journal Article
Characterization of Retinal Structure in ATF6-Associated Achromatopsia
Mastey, Rebecca R, Georgiou, Michalis, Langlo, Christopher S, Kalitzeos, Angelos, Patterson, Emily J, Kane, Thomas, Singh, Navjit, Vincent, Ajoy, Moore, Anthony T, Tsang, Stephen H, Lin, Jonathan H, Young, Marielle P, Hartnett, M Elizabeth, Héon, Elise, Kohl, Susanne, Michaelides, Michel, Carroll, Joseph
Published in Investigative ophthalmology & visual science (01.06.2019)
Published in Investigative ophthalmology & visual science (01.06.2019)
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Journal Article
The Rapid-Onset Chorioretinopathy Phenotype of ABCA4 Disease
Tanaka, Koji, Lee, Winston, Zernant, Jana, Schuerch, Kaspar, Ciccone, Lyam, Tsang, Stephen H., Sparrow, Janet R., Allikmets, Rando
Published in Ophthalmology (Rochester, Minn.) (01.01.2018)
Published in Ophthalmology (Rochester, Minn.) (01.01.2018)
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Journal Article
Proteomic analysis of the human retina reveals region-specific susceptibilities to metabolic- and oxidative stress-related diseases
Velez, Gabriel, Machlab, Daniel A, Tang, Peter H, Sun, Yang, Tsang, Stephen H, Bassuk, Alexander G, Mahajan, Vinit B
Published in PloS one (21.02.2018)
Published in PloS one (21.02.2018)
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Differentiation of hypothalamic-like neurons from human pluripotent stem cells
Wang, Liheng, Meece, Kana, Williams, Damian J, Lo, Kinyui Alice, Zimmer, Matthew, Heinrich, Garrett, Martin Carli, Jayne, Leduc, Charles A, Sun, Lei, Zeltser, Lori M, Freeby, Matthew, Goland, Robin, Tsang, Stephen H, Wardlaw, Sharon L, Egli, Dieter, Leibel, Rudolph L
Published in The Journal of clinical investigation (01.02.2015)
Published in The Journal of clinical investigation (01.02.2015)
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