Genome Editing in Neuroepithelial Stem Cells to Generate Human Neurons with High Adenosine‐Releasing Capacity

Por Poppe, Daniel, Doerr, Jonas, Schneider, Marion, Wilkens, Ruven, Steinbeck, Julius A., Ladewig, Julia, Tam, Allison, Paschon, David E., Gregory, Philip D., Reik, Andreas, Müller, Christa E., Koch, Philipp, Brüstle, Oliver
Publicado no Stem Cells Transl Med (2018)

An Engineered Zinc Finger Protein Activator of the Endogenous Glial Cell Line-Derived Neurotrophic Factor Gene Provides Functional Neuroprotection in a Rat Model of Parkinson's Dis...

Por Laganiere, Josee, Kells, Adrian P., Lai, Jeffrey T., Guschin, Dmitry, Paschon, David E., Meng, Xiangdong, Fong, Lauren K., Yu, Qi, Rebar, Edward J., Gregory, Philip D., Bankiewicz, Krystof S., Forsayeth, John, Zhang, H. Steve
Publicado no J Neurosci (2010)

Mammalian interspecies substitution of immune modulatory alleles by genome editing

Por Lillico, Simon G., Proudfoot, Chris, King, Tim J., Tan, Wenfang, Zhang, Lei, Mardjuki, Rachel, Paschon, David E., Rebar, Edward J., Urnov, Fyodor D., Mileham, Alan J., McLaren, David G., Whitelaw, C. Bruce A.
Publicado no Sci Rep (2016)

Humanized Mouse Model of HIV-1 Latency with Enrichment of Latent Virus in PD-1(+) and TIGIT(+) CD4 T Cells

Por Llewellyn, George N., Seclén, Eduardo, Wietgrefe, Stephen, Liu, Siyu, Chateau, Morgan, Pei, Hua, Perkey, Katherine, Marsden, Matthew D., Hinkley, Sarah J., Paschon, David E., Holmes, Michael C., Zack, Jerome A., Louie, Stan G., Haase, Ashley T., Cannon, Paula M.
Publicado no J Virol (2019)

Efficient targeted gene disruption in the soma and germ line of the frog Xenopus tropicalis using engineered zinc-finger nucleases

Por Young, John J., Cherone, Jennifer M., Doyon, Yannick, Ankoudinova, Irina, Faraji, Farhoud M., Lee, Andrew H., Ngo, Catherine, Guschin, Dmitry Y., Paschon, David E., Miller, Jeffrey C., Zhang, Lei, Rebar, Edward J., Gregory, Philip D., Urnov, Fyodor D., Harland, Richard M., Zeitler, Bryan
Publicado em 2011

LRRK2 mutations cause mitochondrial DNA damage in iPSC-derived neural cells from Parkinson’s disease patients: Reversal by gene correction

Por Sanders, Laurie H., Laganière, Josée, Cooper, Oliver, Mak, Sally K., Vu, B. Joseph, Huang, Y. Anne, Paschon, David E., Vangipuram, Malini, Sundararajan, Ramya, Urnov, Fyodor D., Langston, J. William, Gregory, Philip D., Zhang, H. Steve, Greenamyre, J. Timothy, Isacson, Ole, Schüle, Birgitt
Publicado em 2013

Rapid and efficient clathrin-mediated endocytosis revealed in genome-edited mammalian cells

Por Doyon, Jeffrey B., Zeitler, Bryan, Cheng, Jackie, Cheng, Aaron T., Cherone, Jennifer M., Santiago, Yolanda, Lee, Andrew H., Vo, Thuy D., Doyon, Yannick, Miller, Jeffrey C., Paschon, David E., Zhang, Lei, Rebar, Edward J., Gregory, Philip D., Urnov, Fyodor D., Drubin, David G.
Publicado em 2011

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

Por Sharma, Rajiv, Anguela, Xavier M., Doyon, Yannick, Wechsler, Thomas, DeKelver, Russell C., Sproul, Scott, Paschon, David E., Miller, Jeffrey C., Davidson, Robert J., Shivak, David, Zhou, Shangzhen, Rieders, Julianne, Gregory, Philip D., Holmes, Michael C., Rebar, Edward J., High, Katherine A.
Publicado no Blood (2015)

Non-viral Delivery of Zinc Finger Nuclease mRNA Enables Highly Efficient In Vivo Genome Editing of Multiple Therapeutic Gene Targets

Por Conway, Anthony, Mendel, Matthew, Kim, Kenneth, McGovern, Kyle, Boyko, Alisa, Zhang, Lei, Miller, Jeffrey C., DeKelver, Russell C., Paschon, David E., Mui, Barbara L., Lin, Paulo J.C., Tam, Ying K., Barbosa, Chris, Redelmeier, Tom, Holmes, Michael C., Lee, Gary
Publicado no Mol Ther (2019)

Gene Correction of iPSCs from a Wiskott-Aldrich Syndrome Patient Normalizes the Lymphoid Developmental and Functional Defects

Por Laskowski, Tamara J., Van Caeneghem, Yasmine, Pourebrahim, Rasoul, Ma, Chao, Ni, Zhenya, Garate, Zita, Crane, Ana M., Li, Xuan Shirley, Liao, Wei, Gonzalez-Garay, Manuel, Segovia, Jose Carlos, Paschon, David E., Rebar, Edward J., Holmes, Michael C., Kaufman, Dan, Vandekerckhove, Bart, Davis, Brian R.
Publicado no Stem Cell Reports (2016)

Editing T cell specificity towards leukemia by zinc-finger nucleases and lentiviral gene transfer

Por Provasi, Elena, Genovese, Pietro, Lombardo, Angelo, Magnani, Zulma, Liu, Pei-Qi, Reik, Andreas, Chu, Victoria, Paschon, David E., Zhang, Lei, Kuball, Jurgen, Camisa, Barbara, Bondanza, Attilio, Casorati, Giulia, Ponzoni, Maurilio, Ciceri, Fabio, Bordignon, Claudio, Greenberg, Philip D., Holmes, Michael C., Gregory, Philip D., Naldini, Luigi, Bonini, Chiara
Publicado no Nat Med (2012)

Targeted gene correction of α(1)-antitrypsin deficiency in induced pluripotent stem cells

Por Yusa, Kosuke, Rashid, S. Tamir, Strick-Marchand, Helene, Varela, Ignacio, Liu, Pei-Qi, Paschon, David E., Miranda, Elena, Ordóñez, Adriana, Hannan, Nick, Rouhani, Foad Jafari, Darche, Sylvie, Alexander, Graeme, Marciniak, Stefan J., Fusaki, Noemi, Hasegawa, Mamoru, Holmes, Michael C., Di Santo, James P., Lomas, David A., Bradley, Allan, Vallier, Ludovic
Publicado em 2011

In vivo genome editing restores hemostasis in a mouse model of hemophilia

Por Li, Hojun, Haurigot, Virginia, Doyon, Yannick, Li, Tianjian, Wong, Sunnie Y., Bhagwat, Anand S., Malani, Nirav, Anguela, Xavier M., Sharma, Rajiv, Ivanciu, Lacramiora, Murphy, Samuel L., Finn, Jonathan D., Khazi, Fayaz R., Zhou, Shangzhen, Paschon, David E., Rebar, Edward J., Bushman, Frederic D., Gregory, Philip D., Holmes, Michael C., High, Katherine A.
Publicado em 2011

Targeted Correction and Restored Function of the CFTR Gene in Cystic Fibrosis Induced Pluripotent Stem Cells

Por Crane, Ana M., Kramer, Philipp, Bui, Jacquelin H., Chung, Wook Joon, Li, Xuan Shirley, Gonzalez-Garay, Manuel L., Hawkins, Finn, Liao, Wei, Mora, Daniela, Choi, Sangbum, Wang, Jianbin, Sun, Helena C., Paschon, David E., Guschin, Dmitry Y., Gregory, Philip D., Kotton, Darrell N., Holmes, Michael C., Sorscher, Eric J., Davis, Brian R.
Publicado no Stem Cell Reports (2015)

Diversifying the structure of zinc finger nucleases for high-precision genome editing

Por Paschon, David E., Lussier, Stephanie, Wangzor, Tenzin, Xia, Danny F., Li, Patrick W., Hinkley, Sarah J., Scarlott, Nicholas A., Lam, Stephen C., Waite, Adam J., Truong, Lynn N., Gandhi, Nimisha, Kadam, Bhakti N., Patil, Deepak P., Shivak, David A., Lee, Gary K., Holmes, Michael C., Zhang, Lei, Miller, Jeffrey C., Rebar, Edward J.
Publicado no Nat Commun (2019)

Clinical Scale Zinc Finger Nuclease-mediated Gene Editing of PD-1 in Tumor Infiltrating Lymphocytes for the Treatment of Metastatic Melanoma

Por Beane, Joal D, Lee, Gary, Zheng, Zhili, Mendel, Matthew, Abate-Daga, Daniel, Bharathan, Mini, Black, Mary, Gandhi, Nimisha, Yu, Zhiya, Chandran, Smita, Giedlin, Martin, Ando, Dale, Miller, Jeff, Paschon, David, Guschin, Dmitry, Rebar, Edward J, Reik, Andreas, Holmes, Michael C, Gregory, Philip D, Restifo, Nicholas P, Rosenberg, Steven A, Morgan, Richard A, Feldman, Steven A
Publicado no Mol Ther (2015)

Targeted Gene Addition to a Safe Harbor locus in human CD34(+) Hematopoietic Stem Cells for Correction of X-linked Chronic Granulomatous Disease

Por De Ravin, Suk See, Reik, Andreas, Liu, Pei-Qi, Li, Linhong, Wu, Xiaolin, Su, Ling, Raley, Castle, Theobald, Narda, Choi, Uimook, Song, Alexander H., Chan, Andy, Pearl, Jocelynn R., Paschon, David E., Lee, Janet, Newcombe, Hannah, Koontz, Sherry, Sweeney, Colin, Shivak, David A., Zarember, Kol A., Peshwa, Madhusudan V., Gregory, Philip D., Urnov, Fyodor D., Malech, Harry L.
Publicado no Nat Biotechnol (2016)

Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells

Por Hoban, Megan D., Cost, Gregory J., Mendel, Matthew C., Romero, Zulema, Kaufman, Michael L., Joglekar, Alok V., Ho, Michelle, Lumaquin, Dianne, Gray, David, Lill, Georgia R., Cooper, Aaron R., Urbinati, Fabrizia, Senadheera, Shantha, Zhu, Allen, Liu, Pei-Qi, Paschon, David E., Zhang, Lei, Rebar, Edward J., Wilber, Andrew, Wang, Xiaoyan, Gregory, Philip D., Holmes, Michael C., Reik, Andreas, Hollis, Roger P., Kohn, Donald B.
Publicado no Blood (2015)

Functional footprinting of regulatory DNA

Por Vierstra, Jeff, Reik, Andreas, Chang, Kai-Hsin, Stehling-Sun, Sandra, Zhou, Yuan-Yue, Hinkley, Sarah J., Paschon, David E., Zhang, L., Psatha, Nikoletta, Bendana, Yuri R., O'Neill, Colleen M., Song, Alex H., Mich, Andrea, Liu, Pei-Qi, Lee, Gary, Bauer, Daniel E., Holmes, Michael C., Orkin, Stuart H., Papayannopoulou, Thalia, Stamatoyannopoulos, George, Rebar, Edward J., Gregory, Philip D., Urnov, Fyodor D., Stamatoyannopoulos, John A.
Publicado no Nat Methods (2015)

Functional genomics, proteomics, and regulatory DNA analysis in isogenic settings using zinc finger nuclease-driven transgenesis into a safe harbor locus in the human genome

Por DeKelver, Russell C., Choi, Vivian M., Moehle, Erica A., Paschon, David E., Hockemeyer, Dirk, Meijsing, Sebastiaan H., Sancak, Yasemin, Cui, Xiaoxia, Steine, Eveline J., Miller, Jeffrey C., Tam, Phillip, Bartsevich, Victor V., Meng, Xiangdong, Rupniewski, Igor, Gopalan, Sunita M., Sun, Helena C., Pitz, Kathleen J., Rock, Jeremy M., Zhang, Lei, Davis, Gregory D., Rebar, Edward J., Cheeseman, Iain M., Yamamoto, Keith R., Sabatini, David M., Jaenisch, Rudolf, Gregory, Philip D., Urnov, Fyodor D.
Publicado em 2010