Christian Brendel

United States

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Articles (17)

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2025

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2024

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2024

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2020

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2020

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2020

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2020

Collaborators (12)

Alison Bertuch

Professor

Baylor College of Medicine

UNITED STATES

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Pietro Genovese

Assistant Professor in Pediatrics

Harvard Medical School

UNITED STATES

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Donald Kohn

Professor

University of California, Los Angeles

UNITED STATES

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Kit Shaw

Director, Clinical Translation

Boston Children's Hospital

UNITED STATES

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Alessandro Rambaldi

University of Milan

ITALY

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Mania Ackermann

GERMANY

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Kenneth Cornetta

Professor

Indiana State University

UNITED STATES

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Christopher Bahl

President, Chief Scientific Officer and Co-founder

UNITED STATES

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Guy Sauvageau

Affiliated professor

Université de Montréal

CANADA

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David Dale

Professor of Medicine

University of Washington

UNITED STATES

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Giuseppe Di Caprio

University Of Strathclyde

UNITED KINGDOM

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Emmanuel Payen

INSERM

FRANCE

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Christian Brendel

Research Interests

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Articles (17)

Modeling integration site data for safety assessment with MELISSA

A novel high-titer, bifunctional lentiviral vector for autologous hematopoietic stem cell gene therapy of sickle cell disease

UM171 enhances fitness and engraftment of gene modified hematopoietic stem cells from sickle cells disease patients.

Engineered packaging cell line for the enhanced production of baboon-enveloped retroviral vectors

Epitope editing enables targeted immunotherapy of acute myeloid leukaemia

Genetic reversal of the globin switch concurrently modulates both fetal and sickle hemoglobin and reduces red cell sickling

Swapping the serotype: A novel helper-dependent adenoviral vector platform for in vivo HSC gene therapy

Deletion of murine Rhoh leads to de-repression of Bcl-6 via decreased KAISO levels and accelerates a malignancy phenotype in a murine model of lymphoma

Development of a double shmiR lentivirus effectively targeting both BCL11A and ZNF410 for enhanced induction of fetal hemoglobin to treat β-hemoglobinopathies

Distinct genetic pathways define pre-malignant versus compensatory clonal hematopoiesis in Shwachman-Diamond syndrome.

Restored macrophage function ameliorates disease pathophysiology in a mouse model for IL10 receptor deficient very early onset inflammatory bowel disease.

Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease.

Innovative Therapies for Hemoglobin Disorders.

Current and future gene therapies for hemoglobinopathies

Differential Transgene Silencing of Myeloid-Specific Promoters in the AAVS1 Safe Harbor Locus of Induced Pluripotent Stem Cell-Derived Myeloid Cells

Humanized mice are precious tools for evaluation of hematopoietic gene therapies and preclinical modeling to move towards a clinical trial

Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown for Sickle Cell Gene Therapy

Collaborators (12)

Alison Bertuch

Pietro Genovese

Donald Kohn

Kit Shaw

Alessandro Rambaldi

Mania Ackermann

Kenneth Cornetta

Christopher Bahl

Guy Sauvageau

David Dale

Giuseppe Di Caprio

Emmanuel Payen

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