Gene delivery using AAV8 in vivo for disease stabilization in a bimodal gene therapy approach for the treatment of ADA-deficient SCID

por Carbonaro-Sarracino, Denise A., Chun, Krista, Clark, Danielle N., Kaufman, Michael L., Jin, Xiangyang, Wang, Xiaoyan, Kohn, Donald B.
Publicado en Mol Ther Methods Clin Dev (2021)

Transcriptional profiling of murine retinas undergoing semi-synchronous cone photoreceptor differentiation

por Kaufman, Michael L., Park, Ko Uoon, Goodson, Noah B., Chew, Shereen, Bersie, Stephanie, Jones, Kenneth L., Lamba, Deepak A., Brzezinski, Joseph A.
Publicado en Dev Biol (2019)

Enrichment of Human Hematopoietic Stem/Progenitor Cells Facilitates Transduction for Stem Cell Gene Therapy

por Baldwin, Kismet, Urbinati, Fabrizia, Romero, Zulema, Campo–Fernandez, Beatriz, Kaufman, Michael L., Cooper, Aaron R., Masiuk, Katelyn, Hollis, Roger P., Kohn, Donald B.
Publicado en Stem Cells (2015)

Initiation of Otx2 expression in the developing mouse retina requires a unique enhancer and either Ascl1 or Neurog2 activity

por Kaufman, Michael L., Goodson, Noah B., Park, Ko Uoon, Schwanke, Michael, Office, Emma, Schneider, Sophia R., Abraham, Joy, Hensley, Austin, Jones, Kenneth L., Brzezinski, Joseph A.
Publicado en Development (2021)

Gene therapy/bone marrow transplantation in ADA-deficient mice: roles of enzyme-replacement therapy and cytoreduction

por Carbonaro, Denise A., Jin, Xiangyang, Wang, Xingchao, Yu, Xiao-Jin, Rozengurt, Nora, Kaufman, Michael L., Wang, Xiaoyan, Gjertson, David, Zhou, Yang, Blackburn, Michael R., Kohn, Donald B.
Publicado 2012

Potentially Therapeutic Levels of Anti-Sickling Globin Gene Expression Following Lentivirus-mediated Gene Transfer in Sickle Cell Disease Bone Marrow CD34(+) Cells

por Urbinati, Fabrizia, Hargrove, Philip W., Geiger, Sabine, Romero, Zulema, Wherley, Jennifer, Kaufman, Michael L., Hollis, Roger P., Chambers, Christopher B., Persons, Derek A., Kohn, Donald B., Wilber, Andrew
Publicado en Exp Hematol (2015)

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

por 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
Publicado en Mol Ther (2018)

HSV-sr39TK positron emission tomography and suicide gene elimination of human hematopoietic stem cells and their progeny in humanized mice

por Gschweng, Eric H., McCracken, Melissa N., Kaufman, Michael L., Ho, Michelle, Hollis, Roger P., Wang, Xiaoyan, Saini, Navdeep, Koya, Richard C., Chodon, Thinle, Ribas, Antoni, Witte, Owen N., Kohn, Donald B.
Publicado 2014

The human ankyrin 1 promoter insulator sustains gene expression in a β-globin lentiviral vector in hematopoietic stem cells

por Romero, Zulema, Campo-Fernandez, Beatriz, Wherley, Jennifer, Kaufman, Michael L, Urbinati, Fabrizia, Cooper, Aaron R, Hoban, Megan D, Baldwin, Kismet M, Lumaquin, Dianne, Wang, Xiaoyan, Senadheera, Shantha, Hollis, Roger P, Kohn, Donald B
Publicado en Mol Ther Methods Clin Dev (2015)

Dosing and Re-Administration of Lentiviral Vector for In Vivo Gene Therapy in Rhesus Monkeys and ADA-Deficient Mice

por Carbonaro-Sarracino, Denise A., Tarantal, Alice F., Lee, C. Chang I., Kaufman, Michael L., Wandro, Stephen, Jin, Xiangyang, Martinez, Michele, Clark, Danielle N., Chun, Krista, Koziol, Colin, Hardee, Cinnamon L., Wang, Xiaoyan, Kohn, Donald B.
Publicado en Mol Ther Methods Clin Dev (2019)

Allelic Exclusion and Peripheral Reconstitution by TCR Transgenic T Cells Arising From Transduced Human Hematopoietic Stem/Progenitor Cells

por Giannoni, Francesca, Hardee, Cinnamon L, Wherley, Jennifer, Gschweng, Eric, Senadheera, Shantha, Kaufman, Michael L, Chan, Rebecca, Bahner, Ingrid, Gersuk, Vivian, Wang, Xiaoyan, Gjertson, David, Baltimore, David, Witte, Owen N, Economou, James S, Ribas, Antoni, Kohn, Donald B
Publicado 2013

Improving Gene Editing Outcomes in Human Hematopoietic Stem and Progenitor Cells by Temporal Control of DNA Repair

por Lomova, Anastasia, Clark, Danielle N., Campo-Fernandez, Beatriz, Flores-Bjurström, Carmen, Kaufman, Michael L., Fitz-Gibbon, Sorel, Wang, Xiaoyan, Miyahira, Eric Y., Brown, Devin, DeWitt, Mark A., Corn, Jacob E., Hollis, Roger P., Romero, Zulema, Kohn, Donald B.
Publicado en Stem Cells (2018)

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

por 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.
Publicado en Cell Rep (2018)

Preclinical Demonstration of Lentiviral Vector-mediated Correction of Immunological and Metabolic Abnormalities in Models of Adenosine Deaminase Deficiency

por Carbonaro, Denise A, Zhang, Lin, Jin, Xiangyang, Montiel-Equihua, Claudia, Geiger, Sabine, Carmo, Marlene, Cooper, Aaron, Fairbanks, Lynette, Kaufman, Michael L, Sebire, Neil J, Hollis, Roger P, Blundell, Michael P, Senadheera, Shantha, Fu, Pei-Yu, Sahaghian, Arineh, Chan, Rebecca Y, Wang, Xiaoyan, Cornetta, Kenneth, Thrasher, Adrian J, Kohn, Donald B, Gaspar, H Bobby
Publicado 2014

β-globin gene transfer to human bone marrow for sickle cell disease

por Romero, Zulema, Urbinati, Fabrizia, Geiger, Sabine, Cooper, Aaron R., Wherley, Jennifer, Kaufman, Michael L., Hollis, Roger P., Ruiz de Assin, Rafael, Senadheera, Shantha, Sahagian, Arineh, Jin, Xiangyang, Gellis, Alyse, Wang, Xiaoyan, Gjertson, David, DeOliveira, Satiro, Kempert, Pamela, Shupien, Sally, Abdel-Azim, Hisham, Walters, Mark C., Meiselman, Herbert J., Wenby, Rosalinda B., Gruber, Theresa, Marder, Victor, Coates, Thomas D., Kohn, Donald B.
Publicado 2013

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 en Blood (2015)