Pedro L. Perdigao
Coimbra, Portugal
Last Updated: 09/26/2026
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Found 13 publications
Adenine base editor correction of pathogenic variations associated with inherited retinal dystrophy in patient iPSC and retinal organoids.
Journal: Molecular therapy. Nucleic acids
Published: April 04, 2025
Exploring the potential of cell-derived vesicles for transient delivery of gene editing payloads.
Journal: Advanced drug delivery reviews
Published: December 10, 2023
Effective AAV-mediated gene replacement therapy in retinal organoids modeling AIPL1-associated LCA4.
Effective AAV-mediated gene replacement therapy in retinal organoids modeling AIPL1-associated LCA4.
Journal: Molecular therapy. Nucleic acids
Published: September 05, 2023
A Novel Genetic Variant in MBD5 Associated with Severe Epilepsy and Intellectual Disability: Potential Implications on Neural Primary Cilia.
Journal: International journal of molecular sciences
Published: July 14, 2023
Extracellular communication between brain cells through functional transfer of Cre mRNA mediated by extracellular vesicles.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: February 24, 2023
Extracellular communication between brain cells through functional transfer of Cre mRNA.
Journal: bioRxiv : the preprint server for biology
Published: February 22, 2023
Retinal Organoids from an AIPL1 CRISPR/Cas9 Knockout Cell Line Successfully Recapitulate the Molecular Features of LCA4 Disease.
Journal: International journal of molecular sciences
Published: February 01, 2023
CRISPR-Cas9 correction of a nonsense mutation in LCA5 rescues lebercilin expression and localization in human retinal organoids.
Journal: Molecular therapy. Methods & clinical development
Published: October 11, 2022
Induced Pluripotent Stem Cells and Genome-Editing Tools in Determining Gene Function and Therapy for Inherited Retinal Disorders.
Journal: International journal of molecular sciences
Published: October 04, 2022
Investigation of PTC124-mediated translational readthrough in a retinal organoid model of AIPL1-associated Leber congenital amaurosis.
Journal: Stem cell reports
Published: January 31, 2022
CRISPR-Cas9 correction of OPA1 c.1334G>A: p.R445H restores mitochondrial homeostasis in dominant optic atrophy patient-derived iPSCs.
Journal: Molecular therapy. Nucleic acids
Published: October 28, 2020
Last Updated: 09/26/2026