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List of works by Matthew H Porteus

A Crisper Look at Genome Editing: RNA-guided Genome Modification ⬇️

scientific article published on April 1, 2013

A New Class of Medicines through DNA Editing

scientific article published on 01 March 2019

A survey of ex vivo/in vitro transduction efficiency of mammalian primary cells and cell lines with Nine natural adeno-associated virus (AAV1-9) and one engineered adeno-associated virus serotype ⬇️

scientific article published on March 6, 2013

Author Correction: Engineering monocyte/macrophage-specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing

scientific article published on 20 August 2020

Author Correction: Gene correction for SCID-X1 in long-term hematopoietic stem cells

article

Author Correction: Gene correction for SCID-X1 in long-term hematopoietic stem cells

scientific article published on 04 December 2019

CRISPR-based gene editing enables FOXP3 gene repair in IPEX patient cells

scientific article published on 06 May 2020

Cas9-AAV6-engineered human mesenchymal stromal cells improved cutaneous wound healing in diabetic mice

scientific article published on 18 May 2020

Correction: Multiplexed genetic engineering of human hematopoietic stem and progenitor cells using CRISPR/Cas9 and AAV6

article published in 2018

Design and Development of Artificial Zinc Finger Transcription Factors and Zinc Finger Nucleases to the hTERT Locus ⬇️

scientific article published on April 23, 2013

Development of nuclease-mediated site-specific genome modification ⬇️

scientific article published on September 13, 2012

Diamond Blackfan anemia is mediated by hyperactive Nemo-like kinase

scientific article published on 03 July 2020

Efficient scarless genome editing in human pluripotent stem cells

scientific article published on 30 November 2018

Engineering monocyte/macrophage-specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing

scientific article published on 03 July 2020

Engineering the immune system to cure genetic diseases, HIV, and cancer ⬇️

scientific article published on October 1, 2012

Ethical and regulatory aspects of genome editing.

scientific article published on 6 April 2016

Expanding the Repertoire of Target Sites for Zinc Finger Nuclease-mediated Genome Modification ⬇️

scientific article published on April 30, 2013

Functional significance of U2AF1 S34F mutations in lung adenocarcinomas

scientific article published on 13 December 2019

Gene correction for SCID-X1 in long-term hematopoietic stem cells

article

Gene editing: not just for translation anymore ⬇️

scientific article published on December 28, 2011

Generation of human striatal organoids and cortico-striatal assembloids from human pluripotent stem cells

scientific article published on 03 December 2020

High-efficiency CRISPR induction of t(9;11) chromosomal translocations and acute leukemias in human blood stem cells

scientific article published on 01 October 2019

Homologous recombination‐based gene therapy for the primary immunodeficiencies ⬇️

scientific article published on December 1, 2011

Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I

scientific article published on 06 September 2019

Identification of preexisting adaptive immunity to Cas9 proteins in humans

scientific article published on 28 January 2019

Improving the safety of human pluripotent stem cell therapies using genome-edited orthogonal safeguards

scientific article published on 01 June 2020

Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus.

scientific article published on 8 March 2018

Knock-in editing: it functionally corrects!

scientific article published on May 2016

Metabolic engineering generates a transgene-free safety switch for cell therapy

scientific article published on 13 July 2020

Multiplexed genetic engineering of human hematopoietic stem and progenitor cells using CRISPR/Cas9 and AAV6.

scientific article published on 28 September 2017

Neuronal defects in a human cellular model of 22q11.2 deletion syndrome

scientific article published on 28 September 2020

Newborn screening for severe combined immunodeficiency and T-cell lymphopenia in California: results of the first 2 years

scientific article published on July 2013

Newborn screening for severe combined immunodeficiency in 11 screening programs in the United States

scientific article published in August 2014

Receptor-mediated delivery of engineered nucleases for genome modification ⬇️

scientific article published on August 16, 2013

SCID genotype and 6-month posttransplant CD4 count predict survival and immune recovery

scientific article published on 28 August 2018

Seeing the light: integrating genome engineering with double-strand break repair ⬇️

scientific article published on July 28, 2011

Spatially restricted expression of Dlx-1, Dlx-2 (Tes-1), Gbx-2, and Wnt- 3 in the embryonic day 12.5 mouse forebrain defines potential transverse and longitudinal segmental boundaries ⬇️

scientific article published on July 1, 1993

The TRACE-Seq method tracks recombination alleles and identifies clonal reconstitution dynamics of gene targeted human hematopoietic stem cells

scientific article published on 20 January 2021

Translating the Lessons From Gene Therapy to the Development of Regenerative Medicine ⬇️

scientific article published on March 1, 2011

Transplantation outcomes for severe combined immunodeficiency, 2000-2009.

scientific article

Zinc fingers hit off target ⬇️

scientific article published on October 11, 2011

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