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Molecular Therapy. Advances|May 15, 2026
DNA damage/p53, innate immune, and unfolded protein responses are activated in primate liver after toxic, high-dose AAV-SMN1 deliveryPedram Moeini, Martín Bilbao-Arribas, Elizabeth Guruceaga, et al.
Gene Therapy|March 14, 2026
Building a gene editing lexicon: a model for rare and inherited disordersLeonard A Valentino, Cedric Hermans, Donna Coffin, et al.
Plos One|January 28, 2014
Innate functions of immunoglobulin M lessen liver gene transfer with helper-dependent adenovirusCarmen Unzu, Ignacio Melero, Aizea Morales-Kastresana, et al.
Molecular Therapy. Nucleic Acids|April 6, 2026
Orthogonal characterization of rAAV reveals vector attributes that drive ITR repair, self-complementary genome formation, and transgene expressionCarla Llinares, Suk Namkung, Mitchell Yip, et al.
Hepatology (Baltimore, Md.)|December 15, 2018
Pharmacological Induction of a Progenitor State for the Efficient Expansion of Primary Human HepatocytesCarmen Unzu, Evarist Planet, Nathalie Brandenberg, et al.
Journal of Translational Medicine|June 19, 2012
Transient and intensive pharmacological immunosuppression fails to improve AAV-based liver gene transfer in non-human primatesCarmen Unzu, Sandra Hervás-Stubbs, Ana Sampedro, et al.
Genome Research|June 1, 2014
Loss of transcriptional control over endogenous retroelements during reprogramming to pluripotencyMarc Friedli, Priscilla Turelli, Adamandia Kapopoulou, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|January 21, 2010
Intensive pharmacological immunosuppression allows for repetitive liver gene transfer with recombinant adenovirus in nonhuman primatesAntonio Fontanellas, Sandra Hervás-Stubbs, Itsaso Mauleón, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|September 30, 2010
Sustained enzymatic correction by rAAV-mediated liver gene therapy protects against induced motor neuropathy in acute porphyria miceCarmen Unzu, Ana Sampedro, Itsaso Mauleón, et al.
Molecular Therapy. Nucleic Acids|August 7, 2024
Enhanced AAV transduction across preclinical CNS models: A comparative study in human brain organoids with cross-species evaluationsMatthieu Drouyer, Jessica Merjane, Teodora Nedelkoska, et al.
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