Safety and Efficacy Evaluation for an Ex Vivo Selection Free CRISPR/Cas9 Based Gene Therapy for HAX1 Associated Congenital Neutropenia

被引：0

作者：

Ritter, Malte Ulrich ^{[1
]}

Nasri, Masoud ^{[1
]}

Dannenmann, Benjamin ^{[1
]}

Kaufmann, Masako Monika ^{[2
]}

Zeidler, Karl Alexander ^{[3
]}

Secker, Benjamin ^{[1
]}

Amend, Diana ^{[1
]}

Haaf, Jeremy ^{[1
]}

Zeidler, Cornelia ^{[4
]}

Kliminakou, Maksim ^{[1
]}

Cathomen, Toni ^{[2
]}

Welte, Karl Heinirch ^{[5
]}

Skokowa, Julia ^{[1
]}

机构：

[1] Univ Klinikum Tuebingen, Translat Onkol, Innere Med 2, Tubingen, Germany

[2] Univ Freiburg Klinikum, Inst Transfus Med & Gene Therapy, Freiburg, Germany

[3] Univ Klinikum Tuebingen, Allgemeine Padiatrie Haematol & Onkol, Kinderheilkunde 1, Tubingen, Germany

[4] Hannover Med Sch, Abt Mol Hamatopoese, Klin Padiat Hamatol & Onkol, Hannover, Germany

[5] Univ Klinikum Tuebingen, Mol Haematopoese, Kinderheilkunde 1, Tubingen, Germany

来源：

MOLECULAR THERAPY | 2022年 / 30卷 / 04期

关键词：

D O I：

暂无

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

361

引用

页码：172 / 172

页数：1

共 21 条

[1] A selection free ex vivo gene therapy approach to congenital neutropenia causing HAX1 mutations
Ritter, M. U.
Nasri, M.
Dannenmann, B.
Kaufmann, M. M.
Zeidler, K. A.
Zeidler, C.
Klimiankou, M.
Cathomen, T.
Welte, K.
Skokowa, J.
KLINISCHE PADIATRIE, 2022, 234 (03): : 187 - 187
[2] Efficient Correction of HAX1 Mutations in Primary HSPCs of Severe Congenital Neutropenia Patients Using CRISPR/CAS9 GENE-Editing
Ritter, Malte U.
Secker, Benjamin
Nasri, Masoud
Klimiankou, Maksim
Dannenmann, Benjamin
Amend, Diana
Haaf, Jeremy
Mir, Perihan
Bernhard, Regine
Steiert, Ingeborg
Zeidler, Cornelia
Welte, Karl
Skokowa, Julia
BLOOD, 2020, 136
[3] A selection free Ex vivo gene therapy approach for curing the congenital neutropenia causing HAX1 p.W44X mutation
Ritter, M. U.
Nasri, M.
Zeidler, K. A.
Secker, B.
Dannenmann, B.
Amend, D.
Haaf, J.
Bernhard, R.
Steiert, I
Klimiankou, M.
Zeidler, C.
Welte, K. H.
Skokowa, J.
ONCOLOGY RESEARCH AND TREATMENT, 2021, 44 : 9 - 9
[4] CRISPR/Cas9 system: a powerful technology for in vivo and ex vivo gene therapy
Xiaohui Zhang
Liren Wang
Mingyao Liu
Dali Li
Science China Life Sciences, 2017, 60 : 468 - 475
[5] CRISPR/Cas9 system: a powerful technology for in vivo and ex vivo gene therapy
Xiaohui Zhang
Liren Wang
Mingyao Liu
Dali Li
Science China(Life Sciences), 2017, 60 (05) : 468 - 475
[6] CRISPR/Cas9 system: a powerful technology for in vivo and ex vivo gene therapy
Xiaohui Zhang
Liren Wang
Mingyao Liu
Dali Li
Science China(Life Sciences), 2017, (05) : 468 - 475
[7] CRISPR/Cas9 system: a powerful technology for in vivo and ex vivo gene therapy
Zhang, Xiaohui
Wang, Liren
Liu, Mingyao
Li, Dali
SCIENCE CHINA-LIFE SCIENCES, 2017, 60 (05) : 468 - 475
[8] A Potential CRISPR/Cas9-Based Gene Therapy for Severe Congenital Neutropenia
Ngoc Tung Tran
Van Trung Chu
Kuehn, Ralf
Rajewsky, Klaus
MOLECULAR THERAPY, 2019, 27 (04) : 309 - 310
[9] Gene Therapy Approach for Severe Congenital Neutropenia by CRISPR/Cas9 Gene-Editing of Hematopoietic Stem Cells and iPSCs
Skokowa, Julia
Ritter, Malte
Nasri, Masoud
Mir, Perihan
Dannenmann, Benjamin
Klimiankou, Maksim
Aghaallaei, Nargessadat
Secker, Benjamin
Haaf, Jeremy
Bravo, Natalia Alejandra Borbaran
Zeidler, Cornelia
Bajoghli, Baubak
Xu, Yun
Welte, Karl
MOLECULAR THERAPY, 2020, 28 (04) : 166 - 167
[10] A comparison of different strategies for CRISPR/Cas9-based gene therapies of congenital neutropenia associated with ELANE mutations
Ritter, M. U.
Nasri, M.
Dannenmann, B.
Mir, P.
Secker, B.
Zeidler, C.
Klimiankou, M.
Welte, K.
Skokowa, J.
HUMAN GENE THERAPY, 2024, 35 (3-4) : A175 - A175

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