Latest Publications
Liver gene therapy with intein-mediated F8 trans-splicing corrects mouse haemophilia A
Jun 08, 2022EMBO Molecular Medicine
Read MoreLiver-Directed Adeno-Associated Virus–Mediated Gene Therapy for Mucopolysaccharidosis Type VI
Jun 06, 2022NEJM Evidence
Read MoreThe role of NSP6 in the biogenesis of the SARS-CoV-2 replication organelle
May 13, 2022Nature
Read MoreTherapeutic homology-independent targeted integration in retina and liver
Apr 12, 2022Nature Communications
Read MorePhosphorylation of FAM134C by CK2 controls starvation-induced ER-phagy
Apr 12, 2022Science Advances
Read MoreA single-cell analysis of breast cancer cell lines to study tumour heterogeneity and drug response
Mar 31, 2022Nature Communications
Read MoreModulation of pancreatic cancer cell sensitivity to FOLFIRINOX through microRNA-mediated regulation of DNA damage
Nov 18, 2021Nature Communications
Read MoreComprehensive Analysis of Combinatorial Pharmacological Treatments to Correct Nonsense Mutations in the CFTR Gene
Nov 04, 2021International Journal of Molecular Science
Read MoreCorrection of oxidative stress enhances enzyme replacement therapy in Pompe disease
Oct 26, 2021Embo Molecular Medicine
Read MoreRepurposing of tamoxifen ameliorates CLN3 and CLN7 disease phenotype
Aug 19, 2021EMBO Molecular Medicine
Read MoreSingle-Cell RNA Sequencing Analysis: A Step-by-Step Overview
Jul 30, 2021RNA Bioinformatics. Methods in Molecular Biology, vol 2284. Humana, New York, NY.
Read MoreThe Role of MicroRNAs in Mitochondria-Mediated Eye Diseases
Jun 18, 2021Frontiers in Cell and Developmental Biology
Read MoreAutomatic identification of small molecules that promote cell conversion and reprogramming
May 11, 2021Stem Cell Reports
Read MoreAutomatic synchronisation of the cell cycle in budding yeast through closed-loop feedback control
Apr 27, 2021Nature Communications
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