CRISPR-based gene editing enables FOXP3 gene repair in IPEX patient cells by Goodwin, M, Lee, E, Lakshmanan, U, Shipp, S, Froessl, L, Barzaghi, F, Passerini, L, Narula, M, Sheikali, A, Lee, C M, Bao, G, Bauer, C S, Miller, H K, Garcia-Lloret, M, Butte, M J, Bertaina, A, Shah, A, Pavel-Dinu, M, Hendel, A, Porteus, M, Roncarolo, M G, Bacchetta, R

Get full text

Gene editing using CRISPR enables FOXP3 gene repair in HSPCs and IPEX patient T cells by Lee, E., Goodwin, M., Lakshmanan, U., Shipp, S., Pavel-Dinu, M., Porteus, M., Roncarolo, M., Bacchetta, R.

Get full text

Discovery-based science education: functional genomic dissection in Drosophila by undergraduate researchers by Chen, Jiong, Call, Gerald B, Beyer, Elsa, Bui, Chris, Cespedes, Albert, Chan, Amy, Chan, Jenny, Chan, Stacy, Chhabra, Akanksha, Dang, Peter, Deravanesian, Artemis, Hermogeno, Brenda, Jen, James, Kim, Eunha, Lee, Eric, Lewis, Gemma, Marshall, Jamie, Regalia, Kirsten, Shadpour, Farnaz, Shemmassian, Aram, Spivey, Kristin, Wells, Maggie, Wu, Joy, Yamauchi, Yuki, Yavari, Amir, Abrams, Anna, Abramson, Amanda, Amado, Latiffe, Anderson, Jenny, Bashour, Keenan, Bibikova, Elena, Bookatz, Allen, Brewer, Sarah, Buu, Natalie, Calvillo, Stephanie, Cao, Joseph, Chang, Aileen, Chang, Daniel, Chang, Yuli, Chen, Yibing, Choi, Joo, Chou, Jeyling, Datta, Sumit, Davarifar, Ardy, Desai, Poonam, Fabrikant, Jordan, Farnad, Shahbaz, Fu, Katherine, Garcia, Eddie, Garrone, Nick, Gasparyan, Srpouhi, Gayda, Phyllis, Goffstein, Chad, Gonzalez, Courtney, Guirguis, Mariam, Hassid, Ryan, Hong, Aria, Hong, Julie, Hovestreydt, Lindsay, Hu, Charles, Jamshidian, Farid, Kahen, Katrin, Kao, Linda, Kelley, Melissa, Kho, Thomas, Kim, Sarah, Kim, Yein, Kirkpatrick, Brian, Kohan, Emil, Kwak, Robert, Langenbacher, Adam, Laxamana, Santino, Lee, Chris, Lee, Janet, Lee, So-Youn, Lee, To Hang S, Lee, Toni, Lezcano, Sheila, Lin, Henry, Lin, Peter, Luu, Julie, Luu, Thanh, Marrs, Will, Marsh, Erin, Min, Sarah, Minasian, Tanya, Misra, Amit, Morimoto, Miles, Moshfegh, Yasaman, Murray, Jessica, Nguyen, Cynthia, Nguyen, Kha, Nodado, 2nd, Ernesto, O'Donahue, Amanda, Onugha, Ndidi, Orjiakor, Nneka, Padhiar, Bhavin, Pavel-Dinu, Mara, Pavlenko, Alex, Paz, Edwin

Get full text

Highly Efficient and Marker-free Genome Editing of Human Pluripotent Stem Cells by CRISPR-Cas9 RNP and AAV6 Donor-Mediated Homologous Recombination by Martin, Renata M., Ikeda, Kazuya, Cromer, M. Kyle, Uchida, Nobuko, Nishimura, Toshinobu, Romano, Rosa, Tong, Andrew J., Lemgart, Viktor T., Camarena, Joab, Pavel-Dinu, Mara, Sindhu, Camille, Wiebking, Volker, Vaidyanathan, Sriram, Dever, Daniel P., Bak, Rasmus O., Laustsen, Anders, Lesch, Benjamin J., Jakobsen, Martin R., Sebastiano, Vittorio, Nakauchi, Hiromitsu, Porteus, Matthew H.

Get full text

Gene Editing Rescues In vitro T Cell Development of RAG2-Deficient Induced Pluripotent Stem Cells in an Artificial Thymic Organoid System by Gardner, Cameron L., Pavel-Dinu, Mara, Dobbs, Kerry, Bosticardo, Marita, Reardon, Paul K., Lack, Justin, DeRavin, Suk See, Le, Kent, Bello, Ezekiel, Pala, Francesca, Delmonte, Ottavia M., Malech, Harry, Montel-Hagan, Amelie, Crooks, Gay, Acuto, Oreste, Porteus, Matthew H., Notarangelo, Luigi D.

Get full text

Correction to: Gene Editing Rescues in Vitro T Cell Development of RAG2-Deficient Induced Pluripotent Stem Cells in an Artificial Thymic Organoid System by Gardner, Cameron L., Pavel-Dinu, Mara, Dobbs, Kerry, Bosticardo, Marita, Reardon, Paul K., Lack, Justin, DeRavin, Suk See, Le, Kent, Bello, Ezekiel, Pala, Francesca, Delmonte, Ottavia M., Malech, Harry, Montel-Hagen, Amelie, Crooks, Gay, Acuto, Oreste, Porteus, Matthew H., Notarangelo, Luigi D.

Get full text

High-efficiency transgene integration by homology-directed repair in human primary cells using DNA-PKcs inhibition by Selvaraj, Sridhar, Feist, William N., Viel, Sebastien, Vaidyanathan, Sriram, Dudek, Amanda M., Gastou, Marc, Rockwood, Sarah J., Ekman, Freja K., Oseghale, Aluya R., Xu, Liwen, Pavel-Dinu, Mara, Luna, Sofia E., Cromer, M. Kyle, Sayana, Ruhi, Gomez-Ospina, Natalia, Porteus, Matthew H.

Get full text

Enhanced homology-directed repair for highly efficient gene editing in hematopoietic stem/progenitor cells by De Ravin, Suk See, Brault, Julie, Meis, Ronald J., Liu, Siyuan, Li, Linhong, Pavel-Dinu, Mara, Lazzarotto, Cicera R., Liu, Taylor, Koontz, Sherry M., Choi, Uimook, Sweeney, Colin L., Theobald, Narda, Lee, GaHyun, Clark, Aaron B., Burkett, Sandra S., Kleinstiver, Benjamin P., Porteus, Matthew H., Tsai, Shengdar, Kuhns, Douglas B., Dahl, Gary A., Headey, Stephen, Wu, Xiaolin, Malech, Harry L.

Get full text

Improved Genome Editing through Inhibition of FANCM and Members of the BTR Dissolvase Complex by de Alencastro, Gustavo, Puzzo, Francesco, Pavel-Dinu, Mara, Zhang, Feijie, Pillay, Sirika, Majzoub, Karim, Tiffany, Matthew, Jang, Hagoon, Sheikali, Adam, Cromer, M. Kyle, Meetei, Ruhikanta, Carette, Jan E., Porteus, Matthew H., Pekrun, Katja, Kay, Mark A.

Get full text

Genetically corrected RAG2-SCID human hematopoietic stem cells restore V(D)J-recombinase and rescue lymphoid deficiency by Pavel-Dinu, Mara, Gardner, Cameron L., Nakauchi, Yusuke, Kawai, Tomoki, Delmonte, Ottavia M., Palterer, Boaz, Bosticardo, Marita, Pala, Francesca, Viel, Sebastien, Malech, Harry L., Ghanim, Hana Y., Bode, Nicole M., Kurgan, Gavin L., Detweiler, Angela M., Vakulskas, Christopher A., Neff, Norma F., Sheikali, Adam, Menezes, Sherah T., Chrobok, Jade, Hernández González, Elaine M., Majeti, Ravindra, Notarangelo, Luigi D., Porteus, Matthew H.

Get full text

Survey of Infection Surveillance, Prophylaxis, and Vaccination Practices after Autologous Genetically Modified Cellular Therapies by John, Tami, Marón, Gabriela M., Abraham, Allistair, Bertaina, Alice, Bhoopalan, Senthil Velan, Bidgoli, Alan, Bonfim, Carmem, Coleman, Zane, DeZern, Amy E., Li, Jingjing, Oved, Joseph H., Pavel-Dinu, Mara, Purtill, Duncan, Ruggeri, Annalisa, Russell, Athena, Wynn, Robert F, Boelens, Jaap-Jan, Prockop, Susan E., Sharma, Akshay

Get full text

Genomewide Clonal Analysis of Lethal Mutations in the Drosophila melanogaster Eye: Comparison of the X Chromosome and Autosomes by Villarasa, Nikki, Ngo, Kathy T, Pellegrini, Matteo, Chhabra, Akanksha, Chitsazzadeh, Vida, Fang, Q. Angela, Huang, Cheng R, Kuoy, Edward, Lee, Eric, Lee, JiEun, Lin, Henry, Liu, H-C. Angela, Patananan, Alexander N, Rangel, Alma, Sidhu, Sohrab, Tran, Quynh-Minh, Unkovic, Mary, Wells, Maggie, Zaretsky, Jesse M, An, Ju, Arevalo, Sonia, Ayoub, Danny, Badal, Shawn S, Baghdanian, Arthur H, Cheng, Xibin A, Chin, Mabel, Delosreyes, Angela, Formanes, Arlene J, Kim, Angela Y, Kim, Lydia S-H, Knudsen-Robbins, Chloe, Kohanchi, David, Kovshilovskaya, Bogdana, Landig, Mark E. L, Latterman, Stephanie S, Lei, Kai C, Lin, Kathleen, Makshanoff, Jacob, Picard, Paul D, Salame, Carol, Singh, Inderroop, Song, Junia J, Srivastava, Neetika, Sta.Ana, Jennifer L, Travis, Emily G, Walsh, Thomas, Wu, Ya-Whey, Yu, James S, Bashour, Keenan, Chan, Jenny, Chang, Aileen, Chou, Jeyling, Dang, Peter, Datta, Sumit, Desai, Poonam, Fu, Katherine, Garcia, Eddie, Goffstein, Chad, Guirguis, Mariam, Hassid, Ryan, Hong, Julie, Jamshidian, Farid, Jen, James, Kim, Sarah, Langenbacher, Adam, Lee, So-Youn, Lee, ToHang S, Lewis, Gemma, Lezcano, Sheila, Luu, Julie, Minye, Helena, Misra, Amit, Morimoto, Miles, Moshfegh, Yasaman, Murray, Jessica, Padhiar, Bhavin, Paul, Eric, Pavel-Dinu, Mara, Phaklides, Sarah, Poulose, Preethi, Powell, Russell, Pusic, Aya, Ramola, Divi, Regalia, Kirsten, Saakyan, Manyak, Saarikoski, Pamela, Shadpour, Farnaz, Shemmassian, Aram, Thai, Leo, Tong, Maurine, Tovar, Steve, Trangsrud, Kelly, Tsang, Wah-Yung, Uemura, Marc, Vollmer, Emily, Wood, Damien, Wu, Sophia, Wu, Winston, Yamauchi, Yuki, Yarosh, Will

Get full text