Efficient Correction of ELANE mutations in Primary HSPCs of Severe Congenital Neutropenia Patients Using CRISPR/Cas9 and rAVV6 HDR Repair Templates

被引：3

作者：

Ritter, Malte U. ^{[1
]}

Secker, Benjamin ^{[1
]}

Klimiankou, Maksim ^{[1
]}

Nasri, Masoud ^{[1
]}

Aghaallaei, Narges ^{[1
]}

Dannenmann, Benjamin ^{[1
]}

Bernhard, Regine ^{[1
]}

Steiert, Ingeborg ^{[1
]}

Haehnel, Karin ^{[1
]}

Bajoghli, Baubak ^{[1
]}

Zeidler, Cornelia ^{[2
]}

Welte, Karl ^{[3
]}

Skokowa, Julia ^{[4
]}

机构：

[1] Univ Hosp Tuebingen, Dept Oncol Hematol Immunol Rheumatol & Pulmonol, Tubingen, Germany

[2] Hannover Med Sch, Hematol Oncol, SCNIR, Hannover, Germany

[3] Univ Tubingen, Pediat Hematol & Oncol, Tubingen, Germany

[4] Univ Hosp Tuebingen, Div Translat Oncol, Dept Hematol, Oncol,Clin Immunol, Tubingen, Germany

来源：

BLOOD | 2019年 / 134卷

关键词：

D O I：

10.1182/blood-2019-131708

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

1036

引用

页数：4

共 14 条

[1] 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
[2] CRISPR/Cas9 mediated gene editing of ELANE enables neutrophilic maturation of primary HSPCs and iPSCs of severe congenital neutropenia patients
Nasri, M.
Dannenmann, B.
Ritter, M.
Mir, P.
Amend, D.
Xu, Y.
Klimiankou, M.
Zeidler, C.
Kanz, L.
Welte, K.
Skokowa, J.
ONCOLOGY RESEARCH AND TREATMENT, 2019, 42 : 236 - 236
[3] CRISPR/Cas9 genome editing of hot-spot ELANE mutations in iPSCs of severe Congenital neutropenia patients
Nasri, M.
Dannenmann, B.
Ritter, M.
Xu, Y.
Klimiankou, M.
Zeidler, C.
Lengerke, C.
Welte, K.
Skokowa, J.
ONCOLOGY RESEARCH AND TREATMENT, 2021, 44 : 195 - 195
[4] Disease Modeling of Severe Congenital Neutropenia Using CRISPR/Cas9 Gene Correction or Knockout of ELANE in Patients Derived Induced Pluripotent Stem Cells
Nasri, Masoud
Dannenmann, Benjamin
Mir, Perihan
Ritter, Malte U.
Amend, Diana
Xu, Yun
Klimiankou, Maksim
Zeidler, Cornelia
Welte, Karl
Skokowa, Julia
BLOOD, 2019, 134
[5] CRISPR/Cas9 Strategy for Correcting ELANE Mutations and Restoring Neutrophil Differentiation in Severe Congenital Neutropenia: Insights from Patient-Derived iPSCs
Rydzynska, Zuzanna
Grzela, Dawid
Linder, Monika I.
Dobrewa, Weronika
Urbanska, Zuzanna
Pawlik, Bartlomiej
Krzyzanowski, Damian
Mazurek, Maciej
Janczar, Szymon
Madzio, Joanna
Mlynarski, Wojciech
BLOOD, 2023, 142
[6] CRISPR-Cas9-Mediated ELANE Mutation Correction in Hematopoietic Stem and Progenitor Cells to Treat Severe Congenital Neutropenia
Ngoc Tung Tran
Graf, Robin
Wulf-Goldenberg, Annika
Stecklum, Maria
Strauss, Gabriele
Kuehn, Ralf
Kocks, Christine
Rajewsky, Klaus
Van Trung Chu
MOLECULAR THERAPY, 2020, 28 (12) : 2621 - 2634
[7] Establishment of the Safe and Efficient CRISPR/Cas9-RNP Based Gene-Correction Platform of ELANE Mutations in iPSCs of Severe Congenital Neutropenia (CN) Patients with No Response to G-CSF and High Risk to Develop Leukemia
Nasri, Masoud
Dannenmann, Benjamin
Mir, Perihan
Skroblyn, Tessa
Solovjeva, Anna
Klimiankou, Maksim
Zeidler, Cornelia
Kanz, Lothar
Welte, Karl
Skokowa, Julia
BLOOD, 2017, 130
[8] CRISPR/Cas9-mediated ELANE knockout enables neutrophilic maturation of primary hematopoietic stem and progenitor cells and induced pluripotent stem cells o severe congenital neutropenia patients
Nasri, Masoud
Ritter, Malte
Mir, Perihan
Dannenmann, Benjamin
Aghaallaei, Narges
Amend, Diana
Makaryan, Vahagn
Xu, Yun
Fletcher, Breanna
Bernhard, Regine
Steiert, Ingeborg
Haehnel, Karin
Berger, Juergen
Koch, Iris
Sailer, Brigitte
Hipp, Katharina
Zeidler, Cornelia
Klimiankou, Maksim
Bajoghli, Baubak
Dale, David C.
Welte, Karl
Skokowa, Julia
HAEMATOLOGICA, 2020, 105 (03) : 598 - 609
[9] Correction of X-CGD patient HSPCs by targeted CYBB cDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repair
Colin L. Sweeney
Mara Pavel-Dinu
Uimook Choi
Julie Brault
Taylor Liu
Sherry Koontz
Linhong Li
Narda Theobald
Janet Lee
Ezekiel A. Bello
Xiaolin Wu
Ronald J. Meis
Gary A. Dahl
Matthew H. Porteus
Harry L. Malech
Suk See De Ravin
Gene Therapy, 2021, 28 : 373 - 390
[10] Correction of X-CGD patient HSPCs by targeted CYBB cDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repair
Sweeney, Colin L.
Pavel-Dinu, Mara
Choi, Uimook
Brault, Julie
Liu, Taylor
Koontz, Sherry
Li, Linhong
Theobald, Narda
Lee, Janet
Bello, Ezekiel A.
Wu, Xiaolin
Meis, Ronald J.
Dahl, Gary A.
Porteus, Matthew H.
Malech, Harry L.
De Ravin, Suk See
GENE THERAPY, 2021, 28 (06) : 373 - 390

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