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Narda Theobald

National Institutes of Health

26

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860

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Pre-clinical optimisation and safety studies of a new lentiviral gene therapy for p47phox-deficient chronic granulomatous disease.

2021 Human Gene Therapy
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Enhanced Homology-directed Repair for Highly Efficient Gene Editing in Hematopoietic Stem/Progenitor Cells.

2021 Blood
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Correction of X-CGD patient HSPCs by targeted CYBB cDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repair.

2021 Gene Therapy
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NADPH oxidase correction by mRNA transfection of apheresis granulocytes in chronic granulomatous disease.

2020 Blood Advances
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41. Therapeutic Level CRISPR-Oligomer-Mediated Correction of X-CGD Patient Hematopoietic Stem Cells Using Non-Viral, cGMP Compliant, Scalable, and Closed System

2016 Molecular Therapy
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Lentiviral Hematopoietic Stem Cell Gene Therapy for Older Patients with X-Linked Severe Combined Immunodeficiency

2015 Blood
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Chromothriptic Cure of WHIM Syndrome

2015 Cell
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High Titer Lentivector from a Stable Lenti-Producer Efficiently Corrects CD34+ Hematopoietic Stem Cells from Patients with p47phox-Deficient Chronic Granulomatous Disease

2015 Blood
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Enhancers Are Major Targets for Murine Leukemia Virus Vector Integration

2014 Journal of Virology
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Retrovirus gene therapy for X-linked chronic granulomatous disease can achieve stable long-term correction of oxidase activity in peripheral blood neutrophils.

2010 Blood
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Citations (167)