Francesco Puzzo

About

I am a research scientist skilled in life sciences, cell and molecular biology, and gene therapy. My research aims to develop novel AAV vectors for gene therapy applications, optimizing delivery methods, and studying the efficacy and safety of gene therapies in preclinical models. I look forward to advancing the understanding of gene therapy and developing new and safer therapeutic strategies for a range of genetic and acquired diseases.

Employment

• Telethon Institute Of Genetics And Medicine Researcher
  2026 - Present
• Stanford University Research Scientist
  2024 - 2025
• Stanford University Post doc
  2018 - 2024

Education

• Genethon (France) PhD student
  2014 - 2018
• Università degli Studi di Ferrara Bachelor and Master
  2008 - 2013

Projects & Funding

Projects & funding information is unavailable.

Publications (49)

• Protocol for in vivo DNA-RNA hybrid immunoprecipitation sequencing and analysis from frozen mammalian tissues
  2026 DOI: 10.64898/2026.04.06.716701
  +Save
• Potential effects of endogenous RNA/DNA hybrids on CRISPR-Cas9-mediated homology-directed repair
  Molecular Therapy - Nucleic Acids 2026 DOI: 10.1016/J.OMTN.2026.102880
  +Save
• Tales from the R-loops: how RNA/DNA hybrids may influence CRISPR/Cas9-mediated homologous recombination gene editing outcomes
  2025
  +Save
• The deLIVERed promises of gene therapy: past, present and future of liver-directed gene therapy.
  Molecular therapy : the journal of the American Society of Gene Therapy 2025 DOI: 10.1016/j.ymthe.2025.03.041
  +Save
• AAV-mediated genome editing is influenced by the formation of R-loops.
  Molecular therapy : the journal of the American Society of Gene Therapy 2024 DOI: 10.1016/j.ymthe.2024.09.035
  +Save
• AAV-mediated genome editing is influenced by the formation of R-loops.
  bioRxiv : the preprint server for biology 2024 DOI: 10.1101/2024.05.07.592855
  +Save
• The Efficacy of AAV-Mediated Genome Editing, and Undesired Vector Integrations Are Influenced by R-Loops Formation
  2024
  +Save
• Generation of DNA Aptamers Against Pancreatic Surface Receptors Towards AAV Cell-Specific Targeting
  2023
  +Save
• Promoterless AAV Gene Targeting Vectors Favor Integration into the Albumin Locus Regardless of Homology Arm Sequences
  2023
  +Save
• The Role of DNA/RNA Hybrids on the Efficiency of Nuclease-Free Genome Editing and AAV Integration In Vivo
  2023
  +Save
• Aptamer-programmable adeno-associated viral vectors as a novel platform for cell-specific gene transfer
  Molecular Therapy - Nucleic Acids 2023 DOI: 10.1016/J.OMTN.2023.01.007
  +Save
• Loops as Determinant of AAV Integration by Homologous Recombination
  2022
  +Save
• Programmable Adeno-Associated Viral Vectors for Cell Specific Targeting
  2022
  +Save
• Promoterless AAV Vectors with Homology Arms Can Integrate and Express from Transcriptionally Active Sites in Non-Targeted Loci
  2022
  +Save
• Improved Genome Editing through Inhibition of FANCM and Members of the BTR Dissolvase Complex
  Gustavo de Alencastro, Francesco Puzzo, Mara Pavel-Dinu +12 more
  Molecular Therapy 2021 DOI: 10.1016/J.YMTHE.2020.10.020
  +Save
• Proteins Complex of the Fanconi Anemia Pathway as Determinant of AAV-Mediated Genomic Targeted Integration
  2020
  +Save
• Liver expression of secretable GAA rescues advanced Pompe disease at the biochemical, functional, and transcriptional level in Gaa-/- mice
  Molecular Genetics and Metabolism 2020 DOI: 10.1016/J.YMGME.2019.11.070
  +Save
• Preclinical Development of SPK-3006, an Investigational Liver-Directed AAV Gene Therapy for the Treatment of Pompe Disease
  Molecular Therapy 2020
  +Save
• Preclinical development of SPK-3006, an investigational liver-directed AAV gene therapy for the treatment of Pompe disease
  Molecular Genetics and Metabolism 2020 DOI: 10.1016/J.YMGME.2019.11.029
  +Save
• Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid α-Glucosidase
  Molecular Therapy 2020 DOI: 10.1016/J.YMTHE.2020.05.025
  +Save