Using CRISPR-Cas9 to Generate Gene-Corrected Autologous iPSCs for the Treatment of Inherited Retinal Degeneration
Burnight, Erin R., Gupta, Manav, Wiley, Luke A., Anfinson, Kristin R., Tran, Audrey, Triboulet, Robinson, Hoffmann, Jeremy M., Klaahsen, Darcey L., Andorf, Jeaneen L., Jiao, Chunhua, Sohn, Elliott H., Adur, Malavika K., Ross, Jason W., Mullins, Robert F., Daley, George Q., Schlaeger, Thorsten M., Stone, Edwin M., Tucker, Budd A.
Published in Molecular therapy (06.09.2017)
Published in Molecular therapy (06.09.2017)
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Journal Article
piggyBac transposase tools for genome engineering
Li, Xianghong, Burnight, Erin R, Cooney, Ashley L, Malani, Nirav, Brady, Troy, Sander, Jeffry D, Staber, Janice, Wheelan, Sarah J, Joung, J Keith, McCray, Jr, Paul B, Bushman, Frederic D, Sinn, Patrick L, Craig, Nancy L
Published in Proceedings of the National Academy of Sciences - PNAS (18.06.2013)
Published in Proceedings of the National Academy of Sciences - PNAS (18.06.2013)
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Journal Article
Patient-specific induced pluripotent stem cells (iPSCs) for the study and treatment of retinal degenerative diseases
Wiley, Luke A., Burnight, Erin R., Songstad, Allison E., Drack, Arlene V., Mullins, Robert F., Stone, Edwin M., Tucker, Budd A.
Published in Progress in retinal and eye research (01.01.2015)
Published in Progress in retinal and eye research (01.01.2015)
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Journal Article
cGMP production of patient-specific iPSCs and photoreceptor precursor cells to treat retinal degenerative blindness
Wiley, Luke A., Burnight, Erin R., DeLuca, Adam P., Anfinson, Kristin R., Cranston, Cathryn M., Kaalberg, Emily E., Penticoff, Jessica A., Affatigato, Louisa M., Mullins, Robert F., Stone, Edwin M., Tucker, Budd A.
Published in Scientific reports (29.07.2016)
Published in Scientific reports (29.07.2016)
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Journal Article
Demonstration of the pathogenicity of a common non-exomic mutation in ABCA4 using iPSC-derived retinal organoids and retrospective clinical data
Burnight, Erin R, Fenner, Beau J, Han, Ian C, DeLuca, Adam P, Whitmore, S Scott, Bohrer, Laura R, Andorf, Jeaneen L, Sohn, Elliott H, Mullins, Robert F, Tucker, Budd A, Stone, Edwin M
Published in Human molecular genetics (06.08.2024)
Published in Human molecular genetics (06.08.2024)
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Journal Article
Assessment of Adeno-Associated Virus Serotype Tropism in Human Retinal Explants
Wiley, Luke A, Burnight, Erin R, Kaalberg, Emily E, Jiao, Chunhua, Riker, Megan J, Halder, Jennifer A, Luse, Meagan A, Han, Ian C, Russell, Stephen R, Sohn, Elliott H, Stone, Edwin M, Tucker, Budd A, Mullins, Robert F
Published in Human gene therapy (01.04.2018)
Published in Human gene therapy (01.04.2018)
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Journal Article
Human photoreceptor cells from different macular subregions have distinct transcriptional profiles
Voigt, Andrew P, Mullin, Nathaniel K, Whitmore, S Scott, DeLuca, Adam P, Burnight, Erin R, Liu, Xiuying, Tucker, Budd A, Scheetz, Todd E, Stone, Edwin M, Mullins, Robert F
Published in Human molecular genetics (28.07.2021)
Published in Human molecular genetics (28.07.2021)
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Journal Article
CRISPR-Cas9 genome engineering: Treating inherited retinal degeneration
Burnight, Erin R., Giacalone, Joseph C., Cooke, Jessica A., Thompson, Jessica R., Bohrer, Laura R., Chirco, Kathleen R., Drack, Arlene V., Fingert, John H., Worthington, Kristan S., Wiley, Luke A., Mullins, Robert F., Stone, Edwin M., Tucker, Budd A.
Published in Progress in retinal and eye research (01.07.2018)
Published in Progress in retinal and eye research (01.07.2018)
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Journal Article
Retinal Tropism and Transduction of Adeno-Associated Virus Varies by Serotype and Route of Delivery (Intravitreal, Subretinal, or Suprachoroidal) in Rats
Han, Ian C, Cheng, Justine L, Burnight, Erin R, Ralston, Christy L, Fick, Jessica L, Thomsen, Gabriella J, Tovar, Emilio F, Russell, Stephen R, Sohn, Elliott H, Mullins, Robert F, Stone, Edwin M, Tucker, Budd A, Wiley, Luke A
Published in Human gene therapy (01.12.2020)
Published in Human gene therapy (01.12.2020)
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Journal Article
Patient derived stem cells for discovery and validation of novel pathogenic variants in inherited retinal disease
Mullin, Nathaniel K., Voigt, Andrew P., Cooke, Jessica A., Bohrer, Laura R., Burnight, Erin R., Stone, Edwin M., Mullins, Robert F., Tucker, Budd A.
Published in Progress in retinal and eye research (01.07.2021)
Published in Progress in retinal and eye research (01.07.2021)
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Journal Article
Correction of NR2E3 Associated Enhanced S-cone Syndrome Patient-specific iPSCs using CRISPR-Cas9
Bohrer, Laura R, Wiley, Luke A, Burnight, Erin R, Cooke, Jessica A, Giacalone, Joseph C, Anfinson, Kristin R, Andorf, Jeaneen L, Mullins, Robert F, Stone, Edwin M, Tucker, Budd A
Published in Genes (05.04.2019)
Published in Genes (05.04.2019)
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Journal Article
Helper-Dependent Adenovirus Transduces the Human and Rat Retina but Elicits an Inflammatory Reaction When Delivered Subretinally in Rats
Han, Ian C, Burnight, Erin R, Ulferts, Mallory J, Worthington, Kristan S, Russell, Stephen R, Sohn, Elliott H, Mullins, Robert F, Stone, Edwin M, Tucker, Budd A, Wiley, Luke A
Published in Human gene therapy (01.11.2019)
Published in Human gene therapy (01.11.2019)
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Journal Article
Development and biological characterization of a clinical gene transfer vector for the treatment of MAK-associated retinitis pigmentosa
Tucker, Budd A., Burnight, Erin R., Cranston, Cathryn M., Ulferts, Mallory J., Luse, Meagan A., Westfall, Trudi, Scott, C. Anthony, Marsden, Autumn, Gibson-Corley, Katherine, Wiley, Luke A., Han, Ian C., Slusarski, Diane C., Mullins, Robert F., Stone, Edwin M.
Published in Gene therapy (01.05.2022)
Published in Gene therapy (01.05.2022)
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Journal Article
Development of a Molecularly Stable Gene Therapy Vector for the Treatment of RPGR -Associated X-Linked Retinitis Pigmentosa
Giacalone, Joseph C, Andorf, Jeaneen L, Zhang, Qihong, Burnight, Erin R, Ochoa, Dalyz, Reutzel, Austin J, Collins, Malia M, Sheffield, Val C, Mullins, Robert F, Han, Ian C, Stone, Edwin M, Tucker, Budd A
Published in Human gene therapy (01.08.2019)
Published in Human gene therapy (01.08.2019)
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Journal Article
Using Patient-Specific Induced Pluripotent Stem Cells and Wild-Type Mice to Develop a Gene Augmentation-Based Strategy to Treat CLN3-Associated Retinal Degeneration
Wiley, Luke A, Burnight, Erin R, Drack, Arlene V, Banach, Bailey B, Ochoa, Dalyz, Cranston, Cathryn M, Madumba, Robert A, East, Jade S, Mullins, Robert F, Stone, Edwin M, Tucker, Budd A
Published in Human gene therapy (01.10.2016)
Published in Human gene therapy (01.10.2016)
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Journal Article
Chimeric Helper-Dependent Adenoviruses Transduce Retinal Ganglion Cells and Müller Cells in Human Retinal Explants
Han, Ian C, Burnight, Erin R, Kaalberg, Emily E, Boyce, Timothy M, Stone, Edwin M, Fingert, John H, Mullins, Robert F, Tucker, Budd A, Wiley, Luke A
Published in Journal of ocular pharmacology and therapeutics (01.12.2021)
Published in Journal of ocular pharmacology and therapeutics (01.12.2021)
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Journal Article
Propensity of Patient-Derived iPSCs for Retinal Differentiation: Implications for Autologous Cell Replacement
Cooke, Jessica A, Voigt, Andrew P, Collingwood, Michael A, Stone, Nicholas E, Whitmore, S Scott, DeLuca, Adam P, Burnight, Erin R, Anfinson, Kristin R, Vakulskas, Christopher A, Reutzel, Austin J, Daggett, Heather T, Andorf, Jeaneen L, Stone, Edwin M, Mullins, Robert F, Tucker, Budd A
Published in Stem cells translational medicine (15.06.2023)
Published in Stem cells translational medicine (15.06.2023)
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Journal Article
CRISPRi-Mediated Treatment of Dominant Rhodopsin-Associated Retinitis Pigmentosa
Burnight, Erin R, Wiley, Luke A, Mullin, Nathaniel K, Adur, Malavika K, Lang, Mallory J, Cranston, Cathryn M, Jiao, Chunhua, Russell, Stephen R, Sohn, Elliot H, Han, Ian C, Ross, Jason W, Stone, Edwin M, Mullins, Robert F, Tucker, Budd A
Published in CRISPR journal (01.12.2023)
Published in CRISPR journal (01.12.2023)
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Journal Article
Concise Review: Patient‐Specific Stem Cells to Interrogate Inherited Eye Disease
Giacalone, Joseph C., Wiley, Luke A., Burnight, Erin R., Songstad, Allison E., Mullins, Robert F., Stone, Edwin M., Tucker, Budd A.
Published in Stem cells translational medicine (01.02.2016)
Published in Stem cells translational medicine (01.02.2016)
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Journal Article