J. Bonafont

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.

M. del Río, F. Larcher, M. Escámez, 2022, Matrix biology : journal of the International Society for Matrix Biology.

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

S. Llames, M. del Río, F. Larcher, 2020, Molecular therapy. Methods & clinical development.

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

M. del Río, F. Larcher, Marta García, 2022, Molecular therapy. Methods & clinical development.

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

Wenxin Wang, F. Larcher, Marta García, 2021, Gene Therapy.

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

M. del Río, F. Larcher, Marta García, 2021, Molecular therapy : the journal of the American Society of Gene Therapy.

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

M. del Río, F. Larcher, Marta García, 2019, Molecular therapy : the journal of the American Society of Gene Therapy.

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

F. Larcher, Marta García, Dezhong Zhou, 2021, Gene Therapy.

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

S. Llames, M. del Río, F. Larcher, 2018, Molecular therapy. Nucleic acids.