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Schambach, Axel
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Axel Schambach, Ph.D.
Title
Lecturer on Pediatrics, Part-time
Institution
Boston Children's Hospital
Department
Pediatrics
Address
Children's Hospital
Karp 7 - Williams Lab
300 Longwood Ave
Boston MA 02115
Phone
617/919-2027
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selected publications
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Bibliographic
selected publications
Publications listed below are automatically derived from MEDLINE/PubMed and other sources, which might result in incorrect or missing publications. Faculty can
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to make corrections and additions.
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Field Summary
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Plain Text
PMC Citations
indicate the number of times the publication was cited by articles in PubMed Central, and the
Altmetric
score represents citations in news articles and social media. (Note that publications are often cited in additional ways that are not shown here.)
Fields
are based on how the National Library of Medicine (NLM) classifies the publication's journal and might not represent the specific topic of the publication.
Translation
tags are based on the publication type and the MeSH terms NLM assigns to the publication. Some publications (especially newer ones and publications not in PubMed) might not yet be assigned Field or Translation tags.) Click a Field or Translation tag to filter the publications.
Paasch D, Meyer J, Stamopoulou A, Lenz D, Kuehle J, Kloos D, Buchegger T, Holzinger A, Falk CS, Kloth C, von Kaisenberg CS, Abken H,
Schambach A
, Lachmann N, Morgan M, Moritz T. Ex Vivo Generation of CAR Macrophages from Hematopoietic Stem and Progenitor Cells for Use in Cancer Therapy. Cells. 2022 03 15; 11(6).
PMID:
35326445
.
Citations:
Fields:
Bio
Biophysics
Cel
Cell Biology
Mol
Molecular Biology
Liu Y, Dahl M, Debnath S, Rothe M, Smith EM, Grahn THM, Warsi S, Chen J, Flygare J,
Schambach A
, Karlsson S. Successful gene therapy of Diamond-Blackfan anemia in a mouse model and human CD34+ cord blood hematopoietic stem cells using a clinically applicable lentiviral vector. Haematologica. 2022 02 01; 107(2):446-456.
PMID:
33440921
.
Citations:
3
Fields:
Hem
Hematology
Baron Y, Sens J, Lange L, Nassauer L,
Klatt D
, Hoffmann D, Kleppa MJ,
Barbosa PV
, Keisker M, Steinberg V, Suerth JD, Vondran FWR, Meyer J, Morgan M,
Schambach A
, Galla M. Improved alpharetrovirus-based Gag.MS2 particles for efficient and transient delivery of CRISPR-Cas9 into target cells. Mol Ther Nucleic Acids. 2022 Mar 08; 27:810-823.
PMID:
35141043
.
Citations:
1
Locatelli F, Thompson AA, Kwiatkowski JL, Porter JB, Thrasher AJ, Hongeng S, Sauer MG, Thuret I, Lal A, Algeri M, Schneiderman J, Olson TS, Carpenter B, Amrolia PJ, Anurathapan U,
Schambach A
, Chabannon C, Schmidt M, Labik I, Elliot H, Guo R, Asmal M, Colvin RA, Walters MC. Betibeglogene Autotemcel Gene Therapy for Non-ß0/ß0 Genotype ß-Thalassemia. N Engl J Med. 2022 02 03; 386(5):415-427.
PMID:
34891223
.
Citations:
Fields:
Med
Medicine (General)
Translation:
Humans
Cells
CT
Clinical Trials
Kloos A, Mintzas K, Winckler L, Gabdoulline R, Alwie Y, Jyotsana N, Kattre N, Schottmann R, Scherr M, Gupta C, Adams FF, Schwarzer A, Heckl D,
Schambach A
, Imren S, Humphries RK, Ganser A, Thol F, Heuser M. Correction: Effective drug treatment identified by in vivo screening in a transplantable patient-derived xenograft model of chronic myelomonocytic leukemia. Leukemia. 2021 Dec; 35(12):3629.
PMID:
34728793
.
Citations:
Fields:
Hem
Hematology
Neo
Neoplasms
Rudek LS, Zimmermann K, Galla M, Meyer J, Kuehle J, Stamopoulou A, Brand D, Sandalcioglu IE, Neyazi B, Moritz T, Rossig C, Altvater B, Falk CS, Abken H, Morgan MA,
Schambach A
. Generation of an NF?B-Driven Alpharetroviral "All-in-One" Vector Construct as a Potent Tool for CAR NK Cell Therapy. Front Immunol. 2021; 12:751138.
PMID:
34804035
.
Citations:
Fields:
All
Allergy and Immunology
Translation:
Humans
Cells
Büning H, Fehse B, Ivics Z, Kochanek S, Koehl U, Kupatt C, Mussolino C, Nettelbeck DM,
Schambach A
, Uckert W, Wagner E, Cathomen T. Gene Therapy "Made in Germany": A Historical Perspective, Analysis of the Status Quo, and Recommendations for Action by the German Society for Gene Therapy. Hum Gene Ther. 2021 Oct; 32(19-20):987-996.
PMID:
34662229
.
Citations:
Fields:
Gen
Genetics
The
Therapeutics
Translation:
Humans
Lieske A, Ha TC,
Schambach A
, Maetzig T. An Improved Lentiviral Fluorescent Genetic Barcoding Approach Distinguishes Hematopoietic Stem Cell Properties in Multiplexed In Vivo Experiments. Hum Gene Ther. 2021 10; 32(19-20):1280-1294.
PMID:
34139894
.
Citations:
1
Fields:
Gen
Genetics
The
Therapeutics
Translation:
Animals
Cells
Klapdor R, Wang S, Morgan MA, Zimmermann K, Hachenberg J, Büning H, Dörk T, Hillemanns P,
Schambach A
. NK Cell-Mediated Eradication of Ovarian Cancer Cells with a Novel Chimeric Antigen Receptor Directed against CD44. Biomedicines. 2021 Sep 28; 9(10).
PMID:
34680456
.
Citations:
1
Zhu S, Stanslowsky N, Fernández-Trillo J, Mamo TM, Yu P, Kalmbach N, Ritter B, Eggenschwiler R, Ouwendijk WJD, Mzinza D, Tan L, Leffler A, Spohn M, Brown RJP, Kropp KA, Kaever V, Ha TC, Narayanan P, Grundhoff A, Förster R,
Schambach A
, Verjans GMGM, Schmidt M, Kispert A, Cantz T, Gomis A, Wegner F, Viejo-Borbolla A. Generation of hiPSC-derived low threshold mechanoreceptors containing axonal termini resembling bulbous sensory nerve endings and expressing Piezo1 and Piezo2. Stem Cell Res. 2021 10; 56:102535.
PMID:
34607262
.
Citations:
Fields:
Cel
Cell Biology
Translation:
Humans
Cells
Büning H,
Schambach A
. A first step toward in vivo gene editing in patients. Nat Med. 2021 09; 27(9):1515-1517.
PMID:
34462600
.
Citations:
Fields:
Med
Medicine (General)
Mol
Molecular Biology
Translation:
Humans
Hassan JJ, Lieske A, Dörpmund N,
Klatt D
, Hoffmann D, Kleppa MJ, Kustikova OS, Stahlhut M, Schwarzer A,
Schambach A
, Maetzig T. A Multiplex CRISPR-Screen Identifies PLA2G4A as Prognostic Marker and Druggable Target for HOXA9 and MEIS1 Dependent AML. Int J Mol Sci. 2021 Aug 30; 22(17).
PMID:
34502319
.
Citations:
Fields:
Bio
Biochemistry
Che
Chemistry
Mol
Molecular Biology
Translation:
Humans
Cells
Yang T, Poenisch M, Khanal R, Hu Q, Dai Z, Li R, Song G, Yuan Q, Yao Q, Shen X, Taubert R, Engel B, Jaeckel E, Vogel A, Falk CS,
Schambach A
, Gerovska D, Araúzo-Bravo MJ, Vondran FWR, Cantz T, Horscroft N, Balakrishnan A, Chevessier F, Ott M, Sharma AD. Therapeutic HNF4A mRNA attenuates liver fibrosis in a preclinical model. J Hepatol. 2021 12; 75(6):1420-1433.
PMID:
34453962
.
Citations:
2
Fields:
Gas
Gastroenterology
Translation:
Animals
Schambach A
, Morgan M, Fehse B. Two cases of T cell lymphoma following Piggybac-mediated CAR T cell therapy. Mol Ther. 2021 09 01; 29(9):2631-2633.
PMID:
34433080
.
Citations:
2
Fields:
Mol
Molecular Biology
The
Therapeutics
Translation:
Humans
Cells
Korf-Klingebiel M, Reboll MR, Polten F, Weber N, Jäckle F, Wu X, Kallikourdis M, Kunderfranco P, Condorelli G, Giannitsis E, Kustikova OS,
Schambach A
, Pich A, Widder JD, Bauersachs J, van den Heuvel J, Kraft T, Wang Y, Wollert KC. Myeloid-Derived Growth Factor Protects Against Pressure Overload-Induced Heart Failure by Preserving Sarco/Endoplasmic Reticulum Ca2+-ATPase Expression in Cardiomyocytes. Circulation. 2021 10 12; 144(15):1227-1240.
PMID:
34372689
.
Citations:
Fields:
Car
Cardiology
Vas
Vascular Diseases
Translation:
Humans
Animals
Cells
Morgan MA, Lange L,
Schambach A
. Targeted cytokine delivery: cell therapy to remodel the pre-metastatic niche. Signal Transduct Target Ther. 2021 07 25; 6(1):282.
PMID:
34305139
.
Citations:
Fields:
Cel
Cell Biology
Dru
Drug Therapy
Translation:
Humans
Animals
Janosz E, Hetzel M, Spielmann H, Tumpara S, Rossdam C, Schwabbauer M, Kloos D, von Kaisenberg C,
Schambach A
, Buettner FFR, Janciauskiene S, Lachmann N, Moritz T. Pulmonary transplantation of alpha-1 antitrypsin (AAT)-transgenic macrophages provides a source of functional human AAT in vivo. Gene Ther. 2021 09; 28(9):477-493.
PMID:
34276045
.
Citations:
1
Fields:
Gen
Genetics
The
Therapeutics
Translation:
Humans
Animals
Cells
Morgan MA, Kloos A, Lenz D, Kattre N, Nowak J, Bentele M, Keisker M, Dahlke J, Zimmermann K, Sauer M, Heuser M,
Schambach A
. Improved Activity against Acute Myeloid Leukemia with Chimeric Antigen Receptor (CAR)-NK-92 Cells Designed to Target CD123. Viruses. 2021 07 14; 13(7).
PMID:
34372571
.
Citations:
4
Fields:
Vir
Virology
Translation:
Humans
Animals
Cells
Schwarzer A, Talbot SR, Selich A, Morgan M, Schott JW, Dittrich-Breiholz O, Bastone AL, Weigel B, Ha TC, Dziadek V, Gijsbers R, Thrasher AJ, Staal FJT, Gaspar HB, Modlich U,
Schambach A
, Rothe M. Predicting genotoxicity of viral vectors for stem cell gene therapy using gene expression-based machine learning. Mol Ther. 2021 12 01; 29(12):3383-3397.
PMID:
34174440
.
Citations:
Fields:
Mol
Molecular Biology
The
Therapeutics
Dahlke J, Schott JW, Vollmer Barbosa P,
Klatt D
, Selich A, Lachmann N, Morgan M, Moritz T,
Schambach A
. Efficient Genetic Safety Switches for Future Application of iPSC-Derived Cell Transplants. J Pers Med. 2021 Jun 17; 11(6).
PMID:
34204193
.
Citations:
1
Walcher L, Kistenmacher AK, Sommer C, Böhlen S, Ziemann C, Dehmel S, Braun A, Tretbar US, Klöß S,
Schambach A
, Morgan M, Löffler D, Kämpf C, Blumert C, Reiche K, Beckmann J, König U, Standfest B, Thoma M, Makert GR, Ulbert S, Kossatz-Böhlert U, Köhl U, Dünkel A, Fricke S. Low Energy Electron Irradiation Is a Potent Alternative to Gamma Irradiation for the Inactivation of (CAR-)NK-92 Cells in ATMP Manufacturing. Front Immunol. 2021; 12:684052.
PMID:
34149724
.
Citations:
3
Fields:
All
Allergy and Immunology
Translation:
Humans
Cells
Franzen J, Georgomanolis T, Selich A, Kuo CC, Stöger R, Brant L, Mulabdic MS, Fernandez-Rebollo E, Grezella C, Ostrowska A, Begemann M, Nikolic M, Rath B, Ho AD, Rothe M,
Schambach A
, Papantonis A, Wagner W. DNA methylation changes during long-term in vitro cell culture are caused by epigenetic drift. Commun Biol. 2021 05 19; 4(1):598.
PMID:
34011964
.
Citations:
2
Translation:
Humans
Cells
Schejtman A, Vetharoy W, Choi U, Rivat C, Theobald N, Piras G, Leon-Rico D, Buckland K, Armenteros-Monterroso E, Benedetti S, Ashworth MT, Rothe M,
Schambach A
, Gaspar HB, Kang EM, Malech HL, Thrasher AJ, Santilli G. Preclinical Optimization and Safety Studies of a New Lentiviral Gene Therapy for p47phox-Deficient Chronic Granulomatous Disease. Hum Gene Ther. 2021 09; 32(17-18):949-958.
PMID:
33740872
.
Citations:
Fields:
Gen
Genetics
The
Therapeutics
Translation:
Humans
Animals
Delville M, Bellier F,
Leon J
, Klifa R, Lizot S, Vinçon H, Sobrino S, Thouenon R, Marchal A, Garrigue A, Olivré J, Charbonnier S, Lagresle-Peyrou C, Amendola M,
Schambach A
, Gross D, Lamarthée B,
Benoist C
, Zuber J, André I, Cavazzana M, Six E. A combination of cyclophosphamide and interleukin-2 allows CD4+ T cells converted to Tregs to control scurfy syndrome. Blood. 2021 04 29; 137(17):2326-2336.
PMID:
33545713
.
Citations:
1
Fields:
Hem
Hematology
Translation:
Animals
Cells
Kosanke M, Osetek K, Haase A, Wiehlmann L, Davenport C, Schwarzer A, Adams F, Kleppa MJ,
Schambach A
, Merkert S, Wunderlich S, Menke S, Dorda M, Martin U. Reprogramming enriches for somatic cell clones with small-scale mutations in cancer-associated genes. Mol Ther. 2021 08 04; 29(8):2535-2553.
PMID:
33831558
.
Citations:
2
Fields:
Mol
Molecular Biology
The
Therapeutics
Translation:
Humans
Cells
Morgan MA, Galla M, Grez M, Fehse B,
Schambach A
. Retroviral gene therapy in Germany with a view on previous experience and future perspectives. Gene Ther. 2021 09; 28(9):494-512.
PMID:
33753908
.
Citations:
3
Fields:
Gen
Genetics
The
Therapeutics
Translation:
Humans
Cells
Hoffmann D, Sens J, Brennig S, Brand D, Philipp F, Vollmer Barbosa P, Kuehle J, Steinemann D, Lenz D, Buchegger T, Morgan M, Falk CS, Klein C, Lachmann N,
Schambach A
. Genetic Correction of IL-10RB Deficiency Reconstitutes Anti-Inflammatory Regulation in iPSC-Derived Macrophages. J Pers Med. 2021 Mar 20; 11(3).
PMID:
33804706
.
Citations:
1
Stahlhut M, Ha TC, Takmakova E, Morgan MA, Schwarzer A, Schaudien D, Eder M,
Schambach A
, Kustikova OS. Conditionally immortalised leukaemia initiating cells co-expressing Hoxa9/Meis1 demonstrate microenvironmental adaptation properties ex vivo while maintaining myelomonocytic memory. Sci Rep. 2021 03 05; 11(1):5294.
PMID:
33674652
.
Citations:
Fields:
Sci
Science
Translation:
Humans
Animals
Cells
Lange L, Morgan M,
Schambach A
. The hemogenic endothelium: a critical source for the generation of PSC-derived hematopoietic stem and progenitor cells. Cell Mol Life Sci. 2021 May; 78(9):4143-4160.
PMID:
33559689
.
Citations:
3
Fields:
Cel
Cell Biology
Mol
Molecular Biology
Translation:
Humans
Animals
Cells
Guerrouahen B, Elnaggar M, Al-Mohannadi A, Kizhakayil D, Bonini C, Benjamin R, Brentjens R, Buchholz CJ, Casorati G,
Ferrone S
, Locke FL, Martin F,
Schambach A
, Turtle C, Veys P, van der Vliet HJ, Maccalli C. Proceedings From the First International Workshop at Sidra Medicine: "Engineered Immune Cells in Cancer Immunotherapy (EICCI): From Discovery to Off-the-Shelf Development", 15th-16th February 2019, Doha, Qatar. Front Immunol. 2020; 11:589381.
PMID:
33584653
.
Citations:
Fields:
All
Allergy and Immunology
Translation:
Humans
Animals
Cells
Sens J, Hoffmann D, Lange L, Vollmer Barbosa P, Morgan M, Falk CS,
Schambach A
. Knockout-Induced Pluripotent Stem Cells for Disease and Therapy Modeling of IL-10-Associated Primary Immunodeficiencies. Hum Gene Ther. 2021 01; 32(1-2):77-95.
PMID:
33023317
.
Citations:
3
Fields:
Gen
Genetics
The
Therapeutics
Translation:
Humans
Cells
Moscatelli I, Almarza E,
Schambach A
, Ricks D, Schulz A, Herzog CD, Henriksen K, Askmyr M, Schwartz JD, Richter J. Gene therapy for infantile malignant osteopetrosis: review of pre-clinical research and proof-of-concept for phenotypic reversal. Mol Ther Methods Clin Dev. 2021 Mar 12; 20:389-397.
PMID:
33575431
.
Citations:
1
Ha TC, Stahlhut M, Rothe M, Paul G, Dziadek V, Morgan M, Brugman M, Fehse B, Kustikova O,
Schambach A
, Baum C. Multiple Genes Surrounding Bcl-xL, a Common Retroviral Insertion Site, Can Influence Hematopoiesis Individually or in Concert. Hum Gene Ther. 2021 05; 32(9-10):458-472.
PMID:
33012194
.
Citations:
1
Fields:
Gen
Genetics
The
Therapeutics
Translation:
Humans
Animals
Cells
Dahl M, Smith EMK, Warsi S, Rothe M, Ferraz MJ, Aerts JMFG, Golipour A, Harper C, Pfeifer R, Pizzurro D,
Schambach A
, Mason C, Karlsson S. Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral vector. Mol Ther Methods Clin Dev. 2021 Mar 12; 20:312-323.
PMID:
33511245
.
Citations:
1
Brinkert K, Hedtfeld S, Burhop A, Gastmeier R, Gad P, Wedekind D, Kloth C, Rothschuh J, Lachmann N, Hetzel M, Jirmo AC, Lopez-Rodriguez E, Brandenberger C, Hansen G,
Schambach A
,
Ackermann M
, Tümmler B, Munder A. Rescue from Pseudomonas aeruginosa Airway Infection via Stem Cell Transplantation. Mol Ther. 2021 03 03; 29(3):1324-1334.
PMID:
33279724
.
Citations:
2
Fields:
Mol
Molecular Biology
The
Therapeutics
Translation:
Humans
Animals
Cells
Paredes R, Kelly JR, Geary B, Almarzouq B, Schneider M, Pearson S, Narayanan P, Williamson A, Lovell SC, Wiseman DH, Chadwick JA, Jones NJ, Kustikova O,
Schambach A
, Garner T, Amaral FMR, Pierce A, Stevens A, Somervaille TCP, Whetton AD, Meyer S. EVI1 phosphorylation at S436 regulates interactions with CtBP1 and DNMT3A and promotes self-renewal. Cell Death Dis. 2020 10 20; 11(10):878.
PMID:
33082307
.
Citations:
Fields:
Cel
Cell Biology
Int
Internal Medicine
Translation:
Humans
Cells
Dragon AC, Zimmermann K, Nerreter T, Sandfort D, Lahrberg J, Klöß S, Kloth C, Mangare C, Bonifacius A, Tischer-Zimmermann S, Blasczyk R, Maecker-Kolhoff B, Uchanska-Ziegler B, Abken H,
Schambach A
, Hudecek M, Eiz-Vesper B. CAR-T cells and TRUCKs that recognize an EBNA-3C-derived epitope presented on HLA-B*35 control Epstein-Barr virus-associated lymphoproliferation. J Immunother Cancer. 2020 10; 8(2).
PMID:
33127653
.
Citations:
5
Fields:
Neo
Neoplasms
The
Therapeutics
Translation:
Humans
Klatt D
, Ha TC, Schinke M, Selich A, Lieske A, Dahlke J, Morgan M, Maetzig T,
Schambach A
. Competitive sgRNA Screen Identifies p38 MAPK as a Druggable Target to Improve HSPC Engraftment. Cells. 2020 09 29; 9(10).
PMID:
33003308
.
Citations:
1
Fields:
Bio
Biophysics
Cel
Cell Biology
Mol
Molecular Biology
Translation:
Humans
Animals
Cells
Sgodda M, Alfken S,
Schambach A
, Eggenschwiler R, Fidzinski P, Hummel M, Cantz T. Synthetic Notch-Receptor-Mediated Transmission of a Transient Signal into Permanent Information via CRISPR/Cas9-Based Genome Editing. Cells. 2020 08 20; 9(9).
PMID:
32825374
.
Citations:
Fields:
Bio
Biophysics
Cel
Cell Biology
Mol
Molecular Biology
Translation:
Humans
Morgan MA, Büning H, Sauer M,
Schambach A
. Use of Cell and Genome Modification Technologies to Generate Improved "Off-the-Shelf" CAR T and CAR NK Cells. Front Immunol. 2020; 11:1965.
PMID:
32903482
.
Citations:
23
Fields:
All
Allergy and Immunology
Translation:
Humans
Animals
Cells
Ritter M, Klimiankou M, Klimenkova O,
Schambach A
, Hoffmann D, Schmidt A, Kanz L, Link DC, Welte K, Skokowa J. Cooperating, congenital neutropenia-associated Csf3r and Runx1 mutations activate pro-inflammatory signaling and inhibit myeloid differentiation of mouse HSPCs. Ann Hematol. 2020 Oct; 99(10):2329-2338.
PMID:
32821971
.
Citations:
3
Fields:
Hem
Hematology
Translation:
Animals
Cells
Lange L, Hoffmann D, Schwarzer A, Ha TC, Philipp F, Lenz D, Morgan M,
Schambach A
. Inducible Forward Programming of Human Pluripotent Stem Cells to Hemato-endothelial Progenitor Cells with Hematopoietic Progenitor Potential. Stem Cell Reports. 2020 07 14; 15(1):274.
PMID:
32668220
.
Citations:
1
Fields:
Cel
Cell Biology
Wolfien M,
Klatt D
, Salybekov AA, Ii M, Komatsu-Horii M, Gaebel R, Philippou-Massier J, Schrinner E, Akimaru H, Akimaru E, David R, Garbade J, Gummert J, Haverich A, Hennig H, Iwasaki H, Kaminski A, Kawamoto A, Klopsch C, Kowallick JT, Krebs S, Nesteruk J, Reichenspurner H, Ritter C, Stamm C, Tani-Yokoyama A, Blum H, Wolkenhauer O,
Schambach A
, Asahara T, Steinhoff G. Hematopoietic stem-cell senescence and myocardial repair - Coronary artery disease genotype/phenotype analysis of post-MI myocardial regeneration response induced by CABG/CD133+ bone marrow hematopoietic stem cell treatment in RCT PERFECT Phase 3. EBioMedicine. 2020 Jul; 57:102862.
PMID:
32629392
.
Citations:
7
Fields:
Gen
Genetics
Med
Medicine (General)
Translation:
Humans
Cells
CT
Clinical Trials
Kloos A, Mintzas K, Winckler L, Gabdoulline R, Alwie Y, Jyotsana N, Kattre N, Schottmann R, Scherr M, Gupta C, Adams FF, Schwarzer A, Heckl D,
Schambach A
, Imren S, Humphries RK, Ganser A, Thol F, Heuser M. Effective drug treatment identified by in vivo screening in a transplantable patient-derived xenograft model of chronic myelomonocytic leukemia. Leukemia. 2020 11; 34(11):2951-2963.
PMID:
32576961
.
Citations:
4
Fields:
Hem
Hematology
Neo
Neoplasms
Translation:
Humans
Animals
Jäger B,
Klatt D
, Plappert L, Golpon H, Lienenklaus S, Barbosa PD,
Schambach A
, Prasse A. CXCR4/MIF axis amplifies tumor growth and epithelial-mesenchymal interaction in non-small cell lung cancer. Cell Signal. 2020 09; 73:109672.
PMID:
32428553
.
Citations:
7
Fields:
Cel
Cell Biology
Translation:
Humans
Animals
Cells
Lipus A, Janosz E,
Ackermann M
, Hetzel M, Dahlke J, Buchegger T, Wunderlich S, Martin U, Cathomen T,
Schambach A
, Moritz T, Lachmann N. Targeted Integration of Inducible Caspase-9 in Human iPSCs Allows Efficient in vitro Clearance of iPSCs and iPSC-Macrophages. Int J Mol Sci. 2020 Apr 03; 21(7).
PMID:
32260086
.
Citations:
6
Fields:
Bio
Biochemistry
Che
Chemistry
Mol
Molecular Biology
Translation:
Humans
Cells
Garcia-Perez L, van Eggermond M, van Roon L, Vloemans SA, Cordes M,
Schambach A
, Rothe M, Berghuis D, Lagresle-Peyrou C, Cavazzana M, Zhang F, Thrasher AJ, Salvatori D, Meij P, Villa A, Van Dongen JJM, Zwaginga JJ, van der Burg M, Gaspar HB, Lankester A, Staal FJT, Pike-Overzet K. Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCID. Mol Ther Methods Clin Dev. 2020 Jun 12; 17:666-682.
PMID:
32322605
.
Citations:
11
Brendel C
, Negre O, Rothe M, Guda S, Parsons G, Harris C, McGuinness M, Abriss D, Tsytsykova A,
Klatt D
, Bentler M,
Pellin D
, Christiansen L,
Schambach A
,
Manis J
, Trebeden-Negre H, Bonner M,
Esrick E
, Veres G,
Armant M
,
Williams DA
. Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown for Sickle Cell Gene Therapy. Mol Ther Methods Clin Dev. 2020 Jun 12; 17:589-600.
PMID:
32300607
.
Citations:
9
Hoffmann D, Kuehle J, Lenz D, Philipp F, Zychlinski D, Lachmann N, Moritz T, Steinemann D, Morgan M, Skokowa J, Klein C,
Schambach A
. Lentiviral gene therapy and vitamin B3 treatment enable granulocytic differentiation of G6PC3-deficient induced pluripotent stem cells. Gene Ther. 2020 06; 27(6):297-306.
PMID:
32051561
.
Citations:
2
Fields:
Gen
Genetics
The
Therapeutics
Translation:
Humans
Cells
Zimmermann K, Kuehle J, Dragon AC, Galla M, Kloth C, Rudek LS, Sandalcioglu IE, Neyazi B, Moritz T, Meyer J, Rossig C, Altvater B, Eiz-Vesper B, Morgan MA, Abken H,
Schambach A
. Design and Characterization of an "All-in-One" Lentiviral Vector System Combining Constitutive Anti-GD2 CAR Expression and Inducible Cytokines. Cancers (Basel). 2020 Feb 06; 12(2).
PMID:
32041222
.
Citations:
22
Müller S, Bexte T, Gebel V, Kalensee F, Stolzenberg E, Hartmann J, Koehl U,
Schambach A
, Wels WS, Modlich U, Ullrich E. High Cytotoxic Efficiency of Lentivirally and Alpharetrovirally Engineered CD19-Specific Chimeric Antigen Receptor Natural Killer Cells Against Acute Lymphoblastic Leukemia. Front Immunol. 2019; 10:3123.
PMID:
32117200
.
Citations:
25
Fields:
All
Allergy and Immunology
Translation:
Humans
Cells
Koniaeva E, Stahlhut M, Lange L, Sauer MG, Kustikova OS,
Schambach A
. Conditional Immortalization of Lymphoid Progenitors via Tetracycline-Regulated LMO2 Expression. Hum Gene Ther. 2020 02; 31(3-4):183-198.
PMID:
31760808
.
Citations:
2
Fields:
Gen
Genetics
The
Therapeutics
Translation:
Humans
Animals
Cells
Klatt D
, Cheng E, Hoffmann D, Santilli G, Thrasher AJ,
Brendel C
,
Schambach A
. Differential Transgene Silencing of Myeloid-Specific Promoters in the AAVS1 Safe Harbor Locus of Induced Pluripotent Stem Cell-Derived Myeloid Cells. Hum Gene Ther. 2020 02; 31(3-4):199-210.
PMID:
31773990
.
Citations:
10
Fields:
Gen
Genetics
The
Therapeutics
Translation:
Humans
Cells
Lange L, Hoffmann D, Schwarzer A, Ha TC, Philipp F, Lenz D, Morgan M,
Schambach A
. Inducible Forward Programming of Human Pluripotent Stem Cells to Hemato-endothelial Progenitor Cells with Hematopoietic Progenitor Potential. Stem Cell Reports. 2020 01 14; 14(1):122-137.
PMID:
31839543
.
Citations:
10
Fields:
Cel
Cell Biology
Krooss SA, Dai Z, Schmidt F, Rovai A, Fakhiri J, Dhingra A, Yuan Q, Yang T, Balakrishnan A, Steinbrück L, Srivaratharajan S, Manns MP,
Schambach A
, Grimm D, Bohne J, Sharma AD, Büning H, Ott M. Ex Vivo/In vivo Gene Editing in Hepatocytes Using "All-in-One" CRISPR-Adeno-Associated Virus Vectors with a Self-Linearizing Repair Template. iScience. 2020 Jan 24; 23(1):100764.
PMID:
31887661
.
Citations:
10
Maluski M, Ghosh A, Herbst J, Scholl V, Baumann R, Huehn J, Geffers R, Meyer J, Maul H, Eiz-Vesper B, Krueger A,
Schambach A
, van den Brink MR, Sauer MG. Chimeric antigen receptor-induced BCL11B suppression propagates NK-like cell development. J Clin Invest. 2019 12 02; 129(12):5108-5122.
PMID:
31479431
.
Citations:
8
Fields:
Med
Medicine (General)
Translation:
Humans
Animals
Cells
Selich A, Zimmermann K, Tenspolde M, Dittrich-Breiholz O, von Kaisenberg C,
Schambach A
, Rothe M. Umbilical cord as a long-term source of activatable mesenchymal stromal cells for immunomodulation. Stem Cell Res Ther. 2019 09 23; 10(1):285.
PMID:
31547865
.
Citations:
6
Fields:
Cel
Cell Biology
The
Therapeutics
Translation:
Humans
Animals
Cells
Klatt D
, Cheng E, Philipp F, Selich A, Dahlke J, Schmidt RE, Schott JW, Büning H, Hoffmann D, Thrasher AJ,
Schambach A
. Targeted Repair of p47-CGD in iPSCs by CRISPR/Cas9: Functional Correction without Cleavage in the Highly Homologous Pseudogenes. Stem Cell Reports. 2019 10 08; 13(4):590-598.
PMID:
31543470
.
Citations:
9
Fields:
Cel
Cell Biology
Translation:
Humans
Cells
Charrier S, Lagresle-Peyrou C,
Poletti V
, Rothe M, Cédrone G, Gjata B, Mavilio F, Fischer A,
Schambach A
, de Villartay JP, Cavazzana M, Hacein-Bey-Abina S, Galy A. Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID. Mol Ther Methods Clin Dev. 2019 Dec 13; 15:232-245.
PMID:
31720302
.
Citations:
7
Selich A, Ha TC, Morgan M, Falk CS, von Kaisenberg C,
Schambach A
, Rothe M. Cytokine Selection of MSC Clones with Different Functionality. Stem Cell Reports. 2019 08 13; 13(2):262-273.
PMID:
31303506
.
Citations:
6
Fields:
Cel
Cell Biology
Translation:
Humans
Animals
Cells
Hetzel M, Lopez-Rodriguez E,
Mucci A
, Nguyen AHH, Suzuki T, Shima K, Buchegger T, Dettmer S, Rodt T, Bankstahl JP, Malik P, Knudsen L,
Schambach A
, Hansen G, Trapnell BC, Lachmann N, Moritz T. Effective hematopoietic stem cell-based gene therapy in a murine model of hereditary pulmonary alveolar proteinosis. Haematologica. 2020 04; 105(4):1147-1157.
PMID:
31289207
.
Citations:
2
Fields:
Hem
Hematology
Translation:
Animals
Cells
Löfvall H, Rothe M,
Schambach A
, Henriksen K, Richter J, Moscatelli I. Hematopoietic Stem Cell-Targeted Neonatal Gene Therapy with a Clinically Applicable Lentiviral Vector Corrects Osteopetrosis in oc/oc Mice. Hum Gene Ther. 2019 11; 30(11):1395-1404.
PMID:
31179768
.
Citations:
4
Fields:
Gen
Genetics
The
Therapeutics
Translation:
Humans
Animals
Cells
Schott JW, León-Rico D, Ferreira CB, Buckland KF, Santilli G,
Armant MA
,
Schambach A
, Cavazza A, Thrasher AJ. Enhancing Lentiviral and Alpharetroviral Transduction of Human Hematopoietic Stem Cells for Clinical Application. Mol Ther Methods Clin Dev. 2019 Sep 13; 14:134-147.
PMID:
31338385
.
Citations:
18
Oberschmidt O, Morgan M, Huppert V, Kessler J, Gardlowski T, Matthies N, Aleksandrova K, Arseniev L,
Schambach A
, Koehl U, Kloess S. Development of Automated Separation, Expansion, and Quality Control Protocols for Clinical-Scale Manufacturing of Primary Human NK Cells and Alpharetroviral Chimeric Antigen Receptor Engineering. Hum Gene Ther Methods. 2019 06; 30(3):102-120.
PMID:
30997855
.
Citations:
15
Fields:
Gen
Genetics
The
Therapeutics
Translation:
Humans
Cells
Kloess S, Oberschmidt O, Dahlke J, Vu XK, Neudoerfl C, Kloos A, Gardlowski T, Matthies N, Heuser M, Meyer J, Sauer M, Falk C, Koehl U,
Schambach A
, Morgan MA. Preclinical Assessment of Suitable Natural Killer Cell Sources for Chimeric Antigen Receptor Natural Killer-Based "Off-the-Shelf" Acute Myeloid Leukemia Immunotherapies. Hum Gene Ther. 2019 04; 30(4):381-401.
PMID:
30734584
.
Citations:
17
Fields:
Gen
Genetics
The
Therapeutics
Translation:
Humans
Animals
Cells
Klapdor R, Wang S, Morgan M, Dörk T, Hacker U, Hillemanns P, Büning H,
Schambach A
. Characterization of a Novel Third-Generation Anti-CD24-CAR against Ovarian Cancer. Int J Mol Sci. 2019 Feb 03; 20(3).
PMID:
30717444
.
Citations:
21
Fields:
Bio
Biochemistry
Che
Chemistry
Mol
Molecular Biology
Translation:
Humans
Cells
Ackermann M
, Kempf H, Hetzel M, Hesse C, Hashtchin AR, Brinkert K, Schott JW, Haake K, Kühnel MP, Glage S, Figueiredo C, Jonigk D, Sewald K,
Schambach A
, Wronski S, Moritz T, Martin U, Zweigerdt R, Munder A, Lachmann N. Bioreactor-based mass production of human iPSC-derived macrophages enables immunotherapies against bacterial airway infections. Nat Commun. 2018 11 30; 9(1):5088.
PMID:
30504915
.
Citations:
36
Fields:
Bio
Biology
Sci
Science
Translation:
Humans
Animals
Cells
Poletti V
, Urbinati F, Charrier S, Corre G, Hollis RP, Campo Fernandez B, Martin S, Rothe M,
Schambach A
, Kohn DB, Mavilio F. Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling ßAS3 Globin for Gene Therapy for Sickle Cell Disease. Mol Ther Methods Clin Dev. 2018 Dec 14; 11:167-179.
PMID:
30533448
.
Citations:
6
Morgan MA,
Schambach A
. Engineering CAR-T Cells for Improved Function Against Solid Tumors. Front Immunol. 2018; 9:2493.
PMID:
30420856
.
Citations:
32
Fields:
All
Allergy and Immunology
Translation:
Humans
Cells
Philipp F, Selich A, Rothe M, Hoffmann D, Rittinghausen S, Morgan MA,