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2. Highly Efficient & Specific Repair of MAGT1 Mutation in Xmen Patient T Cells and Hematopoietic Stem Cells

Author

Bzhilyanskaya, Vera, primary, Brault, Julie, additional, Liu, Siyuan, additional, Zea Vera, Andres F., additional, Kozhushko, Nikita, additional, Lawson, Amanda, additional, Choi, Uimook, additional, Clark, Aaron B., additional, Meis, Ronald J., additional, Ma, Michelle, additional, Lazzarotto, Cicera, additional, Tsai, Shengdar, additional, Wu, Xiaolin, additional, Dahl, Gary A., additional, Bergerson, Jenna, additional, Freeman, Alexandra F., additional, Kleinstiver, Benjamin, additional, Malech, Harry L, additional, and De Ravin, Suk See, additional

Published
2022
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3. CRISPR-targeted MAGT1 insertion restores XMEN patient hematopoietic stem cells and lymphocytes

Author

Brault, Julie, primary, Liu, Taylor, additional, Bello, Ezekiel, additional, Liu, Siyuan, additional, Sweeney, Colin L., additional, Meis, Ronald J., additional, Koontz, Sherry, additional, Corsino, Cristina, additional, Choi, Uimook, additional, Vayssiere, Guillaume, additional, Bosticardo, Marita, additional, Dowdell, Kennichi, additional, Lazzarotto, Cicera R., additional, Clark, Aaron B., additional, Notarangelo, Luigi D., additional, Ravell, Juan C., additional, Lenardo, Michael J., additional, Kleinstiver, Benjamin P., additional, Tsai, Shengdar Q., additional, Wu, Xiaolin, additional, Dahl, Gary A., additional, Malech, Harry L., additional, and De Ravin, Suk See, additional

Published
2021
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5. Enhanced homology-directed repair for highly efficient gene editing in hematopoietic stem/progenitor cells

Author

De Ravin, Suk See, primary, Brault, Julie, additional, Meis, Ronald J., additional, Liu, Siyuan, additional, Li, Linhong, additional, Pavel-Dinu, Mara, additional, Lazzarotto, Cicera R., additional, Liu, Taylor, additional, Koontz, Sherry M., additional, Choi, Uimook, additional, Sweeney, Colin L., additional, Theobald, Narda, additional, Lee, GaHyun, additional, Clark, Aaron B., additional, Burkett, Sandra S., additional, Kleinstiver, Benjamin P., additional, Porteus, Matthew H., additional, Tsai, Shengdar, additional, Kuhns, Douglas B., additional, Dahl, Gary A., additional, Headey, Stephen, additional, Wu, Xiaolin, additional, and Malech, Harry L., additional

Published
2021
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6. Enhanced Transduction Lentivector Gene Therapy for Treatment of Older Patients with X-Linked Severe Combined Immunodeficiency

Author

De Ravin, Suk See, primary, Anaya O'Brien, Sandra, primary, Kwatemaa, Nana, primary, Theobald, Narda, primary, Liu, Siyuan, primary, Lee, Janet, primary, Kardava, Lela, primary, Liu, Taylor, primary, Goldman, Frederick, primary, Moir, Susan, primary, Bleesing, Jack, primary, Neven, Benedicte, primary, Puck, Jennifer, primary, Cowan, Morton J, primary, Mamcarz, Ewelina, primary, Gottschalk, Stephen, primary, Meagher, Michael M, primary, Notarangelo, Luigi, primary, Kang, Elizabeth, primary, Wu, Xiaolin, primary, and Malech, Harry L., primary

Published
2019
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8. CRISPR-targeted MAGT1insertion restores XMEN patient hematopoietic stem cells and lymphocytes

Author

Brault, Julie, Liu, Taylor, Bello, Ezekiel, Liu, Siyuan, Sweeney, Colin L., Meis, Ronald J., Koontz, Sherry, Corsino, Cristina, Choi, Uimook, Vayssiere, Guillaume, Bosticardo, Marita, Dowdell, Kennichi, Lazzarotto, Cicera R., Clark, Aaron B., Notarangelo, Luigi D., Ravell, Juan C., Lenardo, Michael J., Kleinstiver, Benjamin P., Tsai, Shengdar Q., Wu, Xiaolin, Dahl, Gary A., Malech, Harry L., and De Ravin, Suk See

Abstract

XMEN disease, defined as “X-linked MAGT1 deficiency with increased susceptibility to Epstein-Barr virus infection and N-linked glycosylation defect,” is a recently described primary immunodeficiency marked by defective T cells and natural killer (NK) cells. Unfortunately, a potentially curative hematopoietic stem cell transplantation is associated with high mortality rates. We sought to develop an ex vivo targeted gene therapy approach for patients with XMEN using a CRISPR/Cas9 adeno-associated vector (AAV) to insert a therapeutic MAGT1gene at the constitutive locus under the regulation of the endogenous promoter. Clinical translation of CRISPR/Cas9 AAV-targeted gene editing (GE) is hampered by low engraftable gene-edited hematopoietic stem and progenitor cells (HSPCs). Here, we optimized GE conditions by transient enhancement of homology-directed repair while suppressing AAV-associated DNA damage response to achieve highly efficient (>60%) genetic correction in engrafting XMEN HSPCs in transplanted mice. Restored MAGT1 glycosylation function in human NK and CD8+T cells restored NK group 2 member D (NKG2D) expression and function in XMEN lymphocytes for potential treatment of infections, and it corrected HSPCs for long-term gene therapy, thus offering 2 efficient therapeutic options for XMEN poised for clinical translation.

Published
2021
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9. CD22 TCR-Engineered T Cells Exert Anti-Leukemia Cytotoxicity without Causing Inflammatory Responses

Author

Ishii, Kazusa, Liu, Zhihui, Davies, John S, Nguyen, Kilyna A, McIntosh, Crystal P, Kadakia, Tejas, Norberg, Scott M, Rae, Zachary, Achar, Sooraj, Altan-Bonnet, Gregoire, Wu, Xiaolin, Kelly, Michael C, Taylor, Naomi, and Hinrichs, Christian S

Abstract

Chimeric antigen receptor (CAR) T cells are remarkably effective, especially against B-cell malignancies. However, CAR-T cell therapy is also associated with cytokine-driven inflammatory toxicities such as cytokine release syndrome. Similar cytokine-related toxicity can occur after bispecific T-cell engager (BiTE) administration. It is unknown whether these inflammatory responses are specifically related to T-cell-mediated therapies that are activated through artificial CAR or BiTE signals.

Published
2023
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10. CD117 Antibody-Drug Conjugate Conditioning Allows Efficient Engraftment of Gene-Modified CD34+ Cells with Fertility Preservation in a Rhesus Gene Therapy Model

Author

Uchida, Naoya, Demirci, Selami, Le, Anh, Chu, Rebecca, Liu, Xiong, Su, Ling, Wu, Xiaolin, Krouse, Allen, Linde, N Seth, Bonifacino, Aylin, Hong, So Gun, Dunbar, Cynthia E., Lanieri, Leanne, Bhat, Anjali, Palchaudhuri, Rahul, Bennet, Bindu, Hoban, Megan D., Bertelsen, Kirk, Olson, Lisa, Donahue, Robert, and Tisdale, John F.

Abstract

Hematopoietic stem cell (HSC) gene therapy is curative for multiple genetic diseases; however, it is limited due to toxicities from chemotherapy-based conditioning. To target HSCs directly, we developed an anti-human CD117 ( c-KIT)-targeted antibody-drug conjugate (CD117-ADC) conjugated to a DNA-binding payload that cross-reacted with non-human primates. To investigate the activity of CD117-ADC in non-human primates, we administered a single injection of ADC without HSC infusion, resulting in a >99% depletion of CD34+CD90+ bone marrow cells in cynomolgus macaques (n=2 of 3 in 0.1 mg/kg and n=3 in 0.3 mg/kg); similar to the ablation observed with 4 doses of myeloablative busulfan (Bu) (n=3). With escalating ADC doses (0.2-0.6 mg/kg), the optimal timing of HSC infusion (6 days in 0.2 or 0.3 mg/kg and 10 days in 0.4mg/kg) was determined in rhesus macaques (n=1 each). The animal dosed 0.6 mg/kg required euthanasia on day 14 due to irreversible hematopoietic suppression and severe liver toxicity. In our preliminary transplantation study, dosing 0.2 mg/kg ADC to rhesus macaques (n=2) resulted in bone marrow depletion and modest-level engraftment of gene-modified cells (vector copy number per cell (VCN) 0.05±0.01) (ASH 2019).

Published
2023
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13. Paired immunoglobulin-like receptor B regulates platelet activation

Author

Fan, Xuemei, primary, Shi, Panlai, additional, Dai, Jing, additional, Lu, Yeling, additional, Chen, Xue, additional, Liu, Xiaoye, additional, Zhang, Kandi, additional, Wu, Xiaolin, additional, Sun, Yueping, additional, Wang, Kemin, additional, Zhu, Li, additional, Zhang, Cheng Cheng, additional, Zhang, Junfeng, additional, Chen, Guo-qiang, additional, Zheng, Junke, additional, and Liu, Junling, additional

Published
2014
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15. The T Cell Receptor As An Oncogene: Thymic Expression Of Self-Reactive T Cell Receptors Targeting Survivin Induces T-Cell Lymphoblastic Leukmia

Author

Cui, Yongzhi, primary, Onozawa, Masahiro, additional, Samsel, Leigh, additional, Garber, Haven, additional, Zhang, Hua, additional, Qin, Haiying, additional, Fry, Terry J, additional, McCoy, J. Philip, additional, Wu, Xiaolin, additional, Aplan, Peter D, additional, and Mackall, Crystal L, additional

Published
2013
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