The 3′ Region of the Chicken Hypersensitive Site-4 Insulator Has Properties Similar to Its Core and Is Required for Full Insulator Activity

Por Arumugam, Paritha I., Urbinati, Fabrizia, Velu, Chinavenmeni S., Higashimoto, Tomoyasu, Grimes, H. Leighton, Malik, Punam
Publicado em 2009

Mechanism of Reduction in Titers From Lentivirus Vectors Carrying Large Inserts in the 3′LTR

Por Urbinati, Fabrizia, Arumugam, Paritha, Higashimoto, Tomoyasu, Perumbeti, Anil, Mitts, Kyle, Xia, Ping, Malik, Punam
Publicado em 2009

A novel human gamma-globin gene vector for genetic correction of sickle cell anemia in a humanized sickle mouse model: critical determinants for successful correction

Por Perumbeti, Ajay, Higashimoto, Tomoyasu, Urbinati, Fabrizia, Franco, Robert, Meiselman, Herbert J., Witte, David, Malik, Punam
Publicado em 2009

Genotoxic Potential of Lineage-specific Lentivirus Vectors Carrying the β-Globin Locus Control Region

Por Arumugam, Paritha I, Higashimoto, Tomoyasu, Urbinati, Fabrizia, Modlich, Ute, Nestheide, Shawna, Xia, Ping, Fox, Catherine, Corsinotti, Andrea, Baum, Christopher, Malik, Punam
Publicado em 2009

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 no Stem Cells (2015)

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 no Exp Hematol (2015)

Reactivating Fetal Hemoglobin Expression in Human Adult Erythroblasts Through BCL11A Knockdown Using Targeted Endonucleases

Por Bjurström, Carmen F, Mojadidi, Michelle, Phillips, John, Kuo, Caroline, Lai, Stephen, Lill, Georgia R, Cooper, Aaron, Kaufman, Michael, Urbinati, Fabrizia, Wang, Xiaoyan, Hollis, Roger P, Kohn, Donald B
Publicado no Mol Ther Nucleic Acids (2016)

Retroviral vector integration deregulates gene expression but has no consequence on the biology and function of transplanted T cells

Por Recchia, Alessandra, Bonini, Chiara, Magnani, Zulma, Urbinati, Fabrizia, Sartori, Daniela, Muraro, Sara, Tagliafico, Enrico, Bondanza, Attilio, Stanghellini, Maria Teresa Lupo, Bernardi, Massimo, Pescarollo, Alessandra, Ciceri, Fabio, Bordignon, Claudio, Mavilio, Fulvio
Publicado em 2006

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 no Mol Ther Methods Clin Dev (2015)

Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy

Por Aiuti, Alessandro, Cassani, Barbara, Andolfi, Grazia, Mirolo, Massimiliano, Biasco, Luca, Recchia, Alessandra, Urbinati, Fabrizia, Valacca, Cristina, Scaramuzza, Samantha, Aker, Memet, Slavin, Shimon, Cazzola, Matteo, Sartori, Daniela, Ambrosi, Alessandro, Di Serio, Clelia, Roncarolo, Maria Grazia, Mavilio, Fulvio, Bordignon, Claudio
Publicado em 2007

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

Por 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
Publicado no Mol Ther (2016)

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 em 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 no Blood (2015)