2022
Aguilar-Gonzalez A. et Al. « Isogenic GAA-KO Murine Muscle Cell Lines Mimicking Severe Pompe Mutations as Preclinical Models for the Screening of Potential Gene Therapy Strategies » International journal of molecular sciences 11, 4-23
Israeli D. et Al.
« Deciphering the Molecular Mechanism of Incurable Muscle Disease by a Novel Method for the Interpretation of miRNA Dysregulation »
Non-coding RNA 8, 48.
Ail D. et Al.
« Systemic and local immune responses to intraocular AAV vector administration in non-human primates »
Molecular therapy Methods & clinical development 24, 306-316
Labasse C. et Al.
« Severe ACTA1-related nemaline myopathy: intranuclear rods, cytoplasmic bodies, and enlarged perinuclear space as characteristic pathological features on muscle biopsies »
Acta neuropathologica communications 10, 101.
Amendola M. et Al.
« CRISPRthripsis: “The Risk of CRISPR/Cas9-induced Chromothripsis in Gene Therapy »
Stem cells translational medicine 11, 1003-1009
