Gene Therapy for the Treatment of Primary Immune Deficiencies

Tekijä Kuo, Caroline Y., Kohn, Donald B.
Julkaisussa Curr Allergy Asthma Rep (2016)

New Frontiers in the Therapy of Primary Immunodeficiency: From Gene Addition to Gene Editing

Tekijä Kohn, Donald B., Kuo, Caroline Y.
Julkaisussa J Allergy Clin Immunol (2017)

Novel Compound Heterozygote Variations in FADD Identified to Cause FAS-Associated Protein with Death Domain Deficiency

Tekijä Kohn, Lisa A., Long, Joseph D., Trope, Edward C., Kuo, Caroline Y.
Julkaisussa J Clin Immunol (2020)

Profound T cell Lymphopenia Associated with Prenatal Exposure to Purine Antagonists Detected by TREC Newborn Screening

Tekijä Kuo, Caroline Y., Garcia-Lloret, Maria I., Slev, Patricia, Bohnsack, John F., Chen, Karin
Julkaisussa J Allergy Clin Immunol Pract (2017)

Adenosine Deaminase (ADA)–Deficient Severe Combined Immune Deficiency (SCID) in the US Immunodeficiency Network (USIDNet) Registry

Tekijä Kuo, Caroline Y., Garabedian, Elizabeth, Puck, Jennifer, Cowan, Morton J., Sullivan, Kathleen E., Buckley, Rebecca H., Cunningham-Rundles, Charlotte, Marsh, Rebecca, Candotti, Fabio, Kohn, Donald B.
Julkaisussa J Clin Immunol (2020)

PTH/PTHrP-SIK3 mediated pathway affects skeletogenesis through altered mTOR signaling

Tekijä Csukasi, Fabiana, Duran, Ivan, Barad, Maya, Barta, Tomas, Gudernova, Iva, Trantirek, Lukas, Martin, Jorge, Kuo, Caroline Y., Woods, Jeremy, Lee, Hane, Cohn, Daniel H., Krejci, Pavel, Krakow, Deborah
Julkaisussa Sci Transl Med (2018)

Characterization of Gene Alterations following Editing of the β-Globin Gene Locus in Hematopoietic Stem/Progenitor Cells

Tekijä Long, Joseph, Hoban, Megan D., Cooper, Aaron R., Kaufman, Michael L., Kuo, Caroline Y., Campo-Fernandez, Beatriz, Lumaquin, Dianne, Hollis, Roger P., Wang, Xiaoyan, Kohn, Donald B., Romero, Zulema
Julkaisussa Mol Ther (2018)

Site-Specific Gene Editing of Human Hematopoietic Stem Cells for X-Linked Hyper-IgM Syndrome

Tekijä Kuo, Caroline Y., Long, Joseph D., Campo-Fernandez, Beatriz, de Oliveira, Satiro, Cooper, Aaron R., Romero, Zulema, Hoban, Megan D., Joglekar, Alok V., Lill, Georgia R., Kaufman, Michael L., Fitz-Gibbon, Sorel, Wang, Xiaoyan, Hollis, Roger P., Kohn, Donald B.
Julkaisussa Cell Rep (2018)

CRISPR/Cas9-Mediated Correction of the Sickle Mutation in Human CD34+ cells

Tekijä Hoban, Megan D, Lumaquin, Dianne, Kuo, Caroline Y, Romero, Zulema, Long, Joseph, Ho, Michelle, Young, Courtney S, Mojadidi, Michelle, Fitz-Gibbon, Sorel, Cooper, Aaron R, Lill, Georgia R, Urbinati, Fabrizia, Campo-Fernandez, Beatriz, Bjurstrom, Carmen F, Pellegrini, Matteo, Hollis, Roger P, Kohn, Donald B
Julkaisussa Mol Ther (2016)

Outcome of domino hematopoietic stem cell transplantation in human subjects: An international case series

Tekijä Belderbos, Mirjam E., Gennery, Andrew R., Dvorak, Christopher C., Blok, Henric-Jan, Eikema, Dirk-Jan, Silva, Juliana M. F., Veys, Paul, Neven, Benedicte, Buckley, Rebecca, Cole, Theresa, Cowan, Morton J., Goebel, W. Scott, Hoenig, Manfred, Kuo, Caroline Y., Stiehm, E. Richard, Wynn, Robert, Bierings, Marc
Julkaisussa J Allergy Clin Immunol (2018)

Editing the Sickle Cell Disease Mutation in Human Hematopoietic Stem Cells: Comparison of Endonucleases and Homologous Donor Templates

Tekijä Romero, Zulema, Lomova, Anastasia, Said, Suzanne, Miggelbrink, Alexandra, Kuo, Caroline Y., Campo-Fernandez, Beatriz, Hoban, Megan D., Masiuk, Katelyn E., Clark, Danielle N., Long, Joseph, Sanchez, Julie M., Velez, Miriam, Miyahira, Eric, Zhang, Ruixue, Brown, Devin, Wang, Xiaoyan, Kurmangaliyev, Yerbol Z., Hollis, Roger P., Kohn, Donald B.
Julkaisussa Mol Ther (2019)

Lentiviral gene therapy for X-linked chronic granulomatous disease

Tekijä Kohn, Donald B., Booth, Claire, Kang, Elizabeth M., Pai, Sung-Yun, Shaw, Kit L., Santilli, Giorgia, Armant, Myriam, Buckland, Karen F., Choi, Uimook, De Ravin, Suk See, Dorsey, Morna J., Kuo, Caroline Y., Leon-Rico, Diego, Rivat, Christine, Izotova, Natalia, Gilmour, Kimberly, Snell, Katie, Dip, Jinhua Xu-Bayford, Darwish, Jinan, Morris, Emma C., Terrazas, Dayna RN, Wang, Leo D., Bauser, Christopher A., Paprotka, Tobias, Kuhns, Douglas B., Gregg, John, Raymond, Hayley E., Everett, John K., Honnet, Geraldine, Biasco, Luca, Newburger, Peter E., Bushman, Frederic D., Grez, Manuel, Gaspar, H. Bobby, Williams, David A., Malech, Harry L., Galy, Anne, Thrasher, Adrian J.
Julkaisussa Nat Med (2020)

Long-term outcomes of 176 patients with X-linked hyper-IgM syndrome treated with or without hematopoietic cell transplantation

Tekijä de la Morena, M. Teresa, Leonard, David, Torgerson, Troy R., Cabral-Marques, Otavio, Slatter, Mary, Aghamohammadi, Asghar, Chandra, Sharat, Murguia-Favela, Luis, Bonilla, Francisco A., Kanariou, Maria, Damrongwatanasuk, Rongras, Kuo, Caroline Y., Dvorak, Christopher C., Meyts, Isabelle, Chen, Karin, Kobrynski, Lisa, Kapoor, Neena, Richter, Darko, DiGiovanni, Daniela, Dhalla, Fatima, Farmaki, Evangelia, Speckmann, Carsten, Español, Teresa, Shcherbina, Anna, Hanson, Imelda Celine, Litzman, Jiri, Routes, John M., Wong, Melanie, Fuleihan, Ramsay, Seneviratne, Suranjith L., Small, Trudy N., Janda, Ales, Bezrodnik, Liliana, Seger, Reinhard, Raccio, Andrea Gomez, Edgar, J. David M., Chou, Janet, Abbott, Jordan K., van Montfrans, Joris, González-Granado, Luis Ignacio, Bunin, Nancy, Kutukculer, Necil, Gray, Paul, Seminario, Gisela, Pasic, Srdjan, Aquino, Victor, Wysocki, Christian, Abolhassani, Hassan, Dorsey, Morna, Cunningham-Rundles, Charlotte, Knutsen, Alan P., Sleasman, John, Carvalho, Beatriz Tavares Costa, Condino-Neto, Antonio, Grunebaum, Eyal, Chapel, Helen, Ochs, Hans D., Filipovich, Alexandra, Cowan, Mort, Gennery, Andrew, Cant, Andrew, Notarangelo, Luigi D., Roifman, Chaim M.
Julkaisussa J Allergy Clin Immunol (2016)