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Journal Articles

High Efficiency Gene Correction in Hematopoietic Cells By Template-Free Crispr/Cas9 Genome Editing

Frank Schnütgen, PhD, Duran Sürün, Joachim Schwäble, PhD, Ana Tomasovic, PhD, Ralf Kühn, PhD, Stefan Stein, PhD, Nina Kurrle, PhD, Hubert Serve, MD, Erhard Seifried, MD PhD, Harald von Melchner, MD PhD

Blood (2016) 128 (22): 3507.

https://doi.org/10.1182/blood.V128.22.3507.3507

Published: 2016

Journal Articles

Phase I/II Gene Therapy Study for Chronic Granulomatous Disease: Results, Lessons and Perspectives.

Blood (2007) 110 (11): 503.

https://doi.org/10.1182/blood.V110.11.503.503

Published: 2007

Journal Articles

The Clonal Inventory of Gene Corrected Hematopoiesis in Three Successful Clinical Gene Therapy Trials

Kerstin Schwarzwaelder, PhD, Manfred Schmidt, PhD, Annette Deichmann, Steven J. Howe, PhD, Marion G. Ott, MD, Stefan Stein, PhD, Ulrich Siler, MD, Salima Hacein-Bey-Abina, PhD, Hanno Glimm, MD, Dieter Hoelzer, MD, Marina Cavazzana-Calvo, MD, PhD, Adrian J. Thrasher, MD, PhD, Reinhard Seger, MD, Manuel Grez, PhD, Christof von Kalle, MD

Blood (2007) 110 (11): 3733.

https://doi.org/10.1182/blood.V110.11.3733.3733

Published: 2007

Journal Articles

Long-Term Clinical Follow-Up and Safety/Toxicity Analysis in 3 X-CGD Patients Treated by Gene Therapy and Non-Myeloablative Conditioning.

Marion G. Ott, MD, Manfred Schmidt, PhD, Stefan Stein, PhD, Kerstin Schwarzwaelder, Ulrich Siler, PhD, Ulrike Koehl, PhD, Klaus Kuehlcke, PhD, Andrea Schilz, PhD, Christoph v. Kalle, PhD, Dieter Hoelzer, MD, Reinhard Seger, MD, Manuel Grez, PhD

Blood (2006) 108 (11): 199.

https://doi.org/10.1182/blood.V108.11.199.199

Published: 2006

Journal Articles

Insertional Activation of MDS1/EVI1, PRDM16 and SETBP1 in a Successful Chronic Granulomatous Disease (CGD) Gene Therapy Trial.

Kerstin Schwarzwaelder, Manfred Schmidt, PhD, Annette Deichmann, Marion G. Ott, MD, Stefan Stein, PhD, Hanno Glimm, MD, Ulrich Siler, MD, Dieter Hoelzer, MD, Reinhard Seger, MD, Manuel Grez, PhD, Christof von Kalle, MD

Blood (2006) 108 (11): 3274.

https://doi.org/10.1182/blood.V108.11.3274.3274

Published: 2006

Journal Articles

Myeloid Expansion after Insertional Activation of MDS1/EVI1, PRDM16 and SETBP1 in a Successful Chronic Granulomatous Gene Therapy Trial.

Kerstin Schwarzwaelder, Manfred Schmidt, PhD, Marion G. Ott, MD, Stefan Stein, PhD, Hanno Glimm, MD, Annette Deichmann, Marlene Fischer, Claudia Ball, Ingo Pilz, Sonja Schmidt, Claudia Prinz, Sylvia Fessler, Manuela Wissler, Silke Klingenberg, Dieter Hoelzer, MD, Reinhard Seger, MD, Manuel Grez, PhD, Christof von Kalle, MD

Blood (2005) 106 (11): 198.

https://doi.org/10.1182/blood.V106.11.198.198

Published: 2005

Journal Articles

Long-Term Follow-Up of Patients Treated by Gene Therapy for X-Linked Chronic Granulomatous Disease.

Marion G. Ott, MD, Manuel Grez, PhD, Stefan Stein, PhD, Ulrich Siler, PhD, Ulrike Koehl, PhD, Klaus Kuehlcke, PhD, Manfred Schmidt, MD, Andrea Schilz, PhD, Hana Kunkel, Sonja Naundorf, PhD, Andrea Brinkmann, Moustapha Hassan, PhD, Christof von Kalle, MD, Adrian Trasher, MD, Reinhard Seger, MD, Dieter Hoelzer, MD

Blood (2005) 106 (11): 194.

https://doi.org/10.1182/blood.V106.11.194.194

Published: 2005

Journal Articles

Gene Therapy for X-Linked Chronic Granulomatous Disease.

Marion G. Ott, MD, Stefan Stein, PhD, Ulrike Koehl, PhD, Andrea Schilz, PhD, Klaus Kuhlcke, PhD, Manfred Schmidt, PhD, Christof von Kalle, MD, Moustapha Hassan, MD, Ulrich Siler, PhD, Dieter Hoelzer, MD, Reinhard Seger, MD, Manuel Grez, PhD

Blood (2004) 104 (11): 409.

https://doi.org/10.1182/blood.V104.11.409.409

Published: 2004

NARROW

High Efficiency Gene Correction in Hematopoietic Cells By Template-Free Crispr/Cas9 Genome Editing

Phase I/II Gene Therapy Study for Chronic Granulomatous Disease: Results, Lessons and Perspectives.

The Clonal Inventory of Gene Corrected Hematopoiesis in Three Successful Clinical Gene Therapy Trials

Long-Term Clinical Follow-Up and Safety/Toxicity Analysis in 3 X-CGD Patients Treated by Gene Therapy and Non-Myeloablative Conditioning.

Insertional Activation of MDS1/EVI1, PRDM16 and SETBP1 in a Successful Chronic Granulomatous Disease (CGD) Gene Therapy Trial.

Myeloid Expansion after Insertional Activation of MDS1/EVI1, PRDM16 and SETBP1 in a Successful Chronic Granulomatous Gene Therapy Trial.

Long-Term Follow-Up of Patients Treated by Gene Therapy for X-Linked Chronic Granulomatous Disease.

Gene Therapy for X-Linked Chronic Granulomatous Disease.

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American Society of Hematology

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ASH Publications

American Society of Hematology

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