Jose Bonafont

Madrid, MD, Spain

Last Updated: 09/26/2026

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Found 11 publications

Proceedings of the second annual meeting of GenE-HumDi (COST Action 21113).

Proceedings of the second annual meeting of GenE-HumDi (COST Action 21113).

Journal: Frontiers in genome editing

Published: July 16, 2025

Safe and Efficacious Permanent Removal of Large COL7A1 Exons for Gene Reframing as a Reliable Therapeutic Strategy for Recessive Dystrophic Epidermolysis Bullosa.

Safe and Efficacious Permanent Removal of Large COL7A1 Exons for Gene Reframing as a Reliable Therapeutic Strategy for Recessive Dystrophic Epidermolysis Bullosa.

Journal: Human gene therapy

Published: May 28, 2025

Harnessing DNA barcoding to enhance and sustain polyclonality in gene-edited hematopoietic stem cells.

Harnessing DNA barcoding to enhance and sustain polyclonality in gene-edited hematopoietic stem cells.

Journal: Molecular therapy. Methods & clinical development

Published: April 07, 2025

Design and validation of a GMP stem cell manufacturing protocol for MPSII hematopoietic stem cell gene therapy.

Design and validation of a GMP stem cell manufacturing protocol for MPSII hematopoietic stem cell gene therapy.

Journal: Molecular therapy. Methods & clinical development

Published: February 05, 2024

Preclinical model for phenotypic correction of dystrophic epidermolysis bullosa by in vivo CRISPR-Cas9 delivery using adenoviral vectors.

Preclinical model for phenotypic correction of dystrophic epidermolysis bullosa by in vivo CRISPR-Cas9 delivery using adenoviral vectors.

Journal: Molecular therapy. Methods & clinical development

Published: January 13, 2022

Mechanistic interrogation of mutation-independent disease modulators of RDEB identifies the small leucine-rich proteoglycan PRELP as a TGF-β antagonist and inhibitor of fibrosis.

Mechanistic interrogation of mutation-independent disease modulators of RDEB identifies the small leucine-rich proteoglycan PRELP as a TGF-β antagonist and inhibitor of fibrosis.

Journal: Matrix biology : journal of the International Society for Matrix Biology

Published: September 30, 2021

Non-viral delivery of CRISPR-Cas9 complexes for targeted gene editing via a polymer delivery system.

Non-viral delivery of CRISPR-Cas9 complexes for targeted gene editing via a polymer delivery system.

Journal: Gene therapy

Published: August 19, 2020

Correction of recessive dystrophic epidermolysis bullosa by homology-directed repair-mediated genome editing.

Correction of recessive dystrophic epidermolysis bullosa by homology-directed repair-mediated genome editing.

Journal: Molecular therapy : the journal of the American Society of Gene Therapy

Published: August 08, 2020

Efficient CRISPR-Cas9-Mediated Gene Ablation in Human Keratinocytes to Recapitulate Genodermatoses: Modeling of Netherton Syndrome.

Efficient CRISPR-Cas9-Mediated Gene Ablation in Human Keratinocytes to Recapitulate Genodermatoses: Modeling of Netherton Syndrome.

Journal: Molecular therapy. Methods & clinical development

Published: February 07, 2020

Clinically Relevant Correction of Recessive Dystrophic Epidermolysis Bullosa by Dual sgRNA CRISPR/Cas9-Mediated Gene Editing.

Clinically Relevant Correction of Recessive Dystrophic Epidermolysis Bullosa by Dual sgRNA CRISPR/Cas9-Mediated Gene Editing.

Journal: Molecular therapy : the journal of the American Society of Gene Therapy

Published: October 01, 2018

Deletion of a Pathogenic Mutation-Containing Exon of COL7A1 Allows Clonal Gene Editing Correction of RDEB Patient Epidermal Stem Cells.

Deletion of a Pathogenic Mutation-Containing Exon of COL7A1 Allows Clonal Gene Editing Correction of RDEB Patient Epidermal Stem Cells.

Journal: Molecular therapy. Nucleic acids

Published: December 22, 2017

Showing 1-11 of 11

Last Updated: 09/26/2026