2024
TNFα prevents FGF4-mediated rescue of astrocyte dysfunction and reactivity in human ALS models
Neurobiology of Disease. 2024. Vol. 201. DOI : 10.1016/j.nbd.2024.106687.Single-cell and spatial atlases of spinal cord injury in the Tabulae Paralytica
Nature. 2024. Vol. 631, num. 8019, p. 150 – 163. DOI : 10.1038/s41586-024-07504-y.Production and Purification of Adeno-Associated Viral Vectors (AAVs) Using Orbitally Shaken HEK293 Cells
Recombinant Protein Expression in Mammalian Cells; Humana Press, 2024. p. 55 – 74.2023
Long term peripheral AAV9-SMN gene therapy promotes survival in a mouse model of spinal muscular atrophy
Human Molecular Genetics. 2023. DOI : 10.1093/hmg/ddad202.Stable isotope labeling and ultra-high-resolution NanoSIMS imaging reveal alpha-synuclein-induced changes in neuronal metabolism in vivo
Acta Neuropathologica Communications. 2023. Vol. 11, num. 1, p. 157. DOI : 10.1186/s40478-023-01608-8.AAV9-mediated SMN gene therapy rescues cardiac desmin but not lamin A/C and elastin dysregulation in Smn(2B/-) spinal muscular atrophy mice
Human Molecular Genetics. 2023. DOI : 10.1093/hmg/ddad121.The role of the NF-kappaB pathway in Amyotrophic lateral sclerosis pathogenesis
Lausanne, EPFL, 2023.2022
Gene therapy against amyotrophic lateral sclerosis: a bicistronic AAV vector for RNAi against mutated SOD1
2022. 29th Annual Congress of the European-Society-of-Gene-and-Cell-Therapy (ESCGT), Edinburgh, SCOTLAND, Oct 11-14, 2022. p. A89 – A89.Central and peripheral delivered AAV9-SMN are both efficient but target different pathomechanisms in a mouse model of spinal muscular atrophy
Gene Therapy. 2022. DOI : 10.1038/s41434-022-00338-1.Astrocyte-targeting RNA interference against mutated superoxide dismutase 1 induces motoneuron plasticity and protects fast-fatigable motor units in a mouse model of amyotrophic lateral sclerosis
Glia. 2022. DOI : 10.1002/glia.24140.Dopamine and Methamphetamine Differentially Affect Electron Transport Chain Complexes and Parkin in Rat Striatum: New Insight into Methamphetamine Neurotoxicity
International Journal Of Molecular Sciences. 2022. Vol. 23, num. 1, p. 363. DOI : 10.3390/ijms23010363.2021
The exercise-induced long noncoding RNA CYTOR promotes fast-twitch myogenesis in aging
Science Translational Medicine. 2021. Vol. 13, num. 623, p. eabc7367. DOI : 10.1126/scitranslmed.abc7367.Proteins associated with the sarcomere and costamere in hearts are dysregulated in two mouse models of spinal muscular atrophy
2021. p. S131 – S132. DOI : 10.1016/j.nmd.2021.07.295.CSP alpha reduces aggregates and rescues striatal dopamine release in alpha-synuclein transgenic mice
Brain. 2021. Vol. 144, p. 1661 – 1669. DOI : 10.1093/brain/awab076.Parkin regulates drug-taking behavior in rat model of methamphetamine use disorder
Translational Psychiatry. 2021. Vol. 11, num. 1, p. 293. DOI : 10.1038/s41398-021-01387-7.IL10-and IL35-Secreting MutuDC Lines Act in Cooperation to Inhibit Memory T Cell Activation Through LAG-3 Expression
Frontiers In Immunology. 2021. Vol. 12, p. 607315. DOI : 10.3389/fimmu.2021.607315.SMN Depleted Mice Offer a Robust and Rapid Onset Model of Nonalcoholic Fatty Liver Disease
Cellular And Molecular Gastroenterology And Hepatology. 2021. Vol. 12, num. 1, p. 354 – +. DOI : 10.1016/j.jcmgh.2021.01.019.Mitofusin-2 in nucleus accumbens regulates anxiety and depression-like behaviors through mitochondrial and neuronal actions
Biological Psychiatry. 2021. Vol. 89, num. 11, p. 1024 – 1026. DOI : 10.1016/j.biopsych.2020.12.003.2020
Anti-Aβ antibodies bound to neuritic plaques enhance microglia activity and mitigate tau pathology
Acta Neuropathologica Communications. 2020. Vol. 8, num. 1, p. 198. DOI : 10.1186/s40478-020-01069-3.Efficient viral transduction in mouse inner ear hair cells with utricle injection and AAV9-PHP.B
Hearing Research. 2020. Vol. 394, p. 107882. DOI : 10.1016/j.heares.2020.107882.Nano-imaging trace elements at organelle levels in substantia nigra overexpressing alpha-synuclein to model Parkinson’s disease
Communications Biology. 2020. Vol. 3, num. 1, p. 364. DOI : 10.1038/s42003-020-1084-0.Glutaredoxin 1 Downregulation in the Substantia Nigra Leads to Dopaminergic Degeneration in Mice
Movement Disorders. 2020. Vol. 45, num. 10, p. 1843 – 1853. DOI : 10.1002/mds.28190.Blood Flow to the Spleen is Altered in a Mouse Model of Spinal Muscular Atrophy
Journal Of Neuromuscular Diseases. 2020. Vol. 7, num. 3, p. 315 – 322. DOI : 10.3233/JND-200493.2019
Glutaredoxin1 Diminishes Amyloid Beta-Mediated Oxidation of F-Actin and Reverses Cognitive Deficits in an Alzheimer’s Disease Mouse Model
Antioxidants & Redox Signaling. 2019. Vol. 31, num. 18, p. 1321 – 1338. DOI : 10.1089/ars.2019.7754.Allele-specific gene disruption through discrimination of a single base change by S. aureus Cas9-KKH prevents progressive hearing loss after AAV-mediated gene delivery
2019. ESGCT 27th Annual Congress in collaboration with SETGyc Meeting, Barcelona, SPAIN, Oct 22-25, 2019. p. A11 – A12.Delivery of CRISPR/Cas9 using AAV-PHP.B in the inner ear leads to allele-specific inactivation of the mutated Tmc1 allele and protects auditory function in Beethoven mice
2019. ESGCT 27th Annual Congress in collaboration with SETGyc Meeting, Barcelona, SPAIN, Oct 22-25, 2019. p. A106 – A107.Allele-specific gene editing prevents deafness in a model of dominant progressive hearing loss
Nature Medicine. 2019. Vol. 25, num. 7, p. 1123 – 1130. DOI : 10.1038/s41591-019-0500-9.Evolution of the neurochemical profiles in the G93A-SOD1 mouse model of amyotrophic lateral sclerosis
Journal of Cerebral Blood Flow and Metabolism. 2019. Vol. 39, num. 7, p. 1283 – 1298. DOI : 10.1177/0271678X18756499.Scalable Production of AAV Vectors in Orbitally Shaken HEK293 Cells
Molecular Therapy-Methods & Clinical Development. 2019. Vol. 13, p. 14 – 26. DOI : 10.1016/j.omtm.2018.11.004.The RNA-Binding Protein PUM2 Impairs Mitochondrial Dynamics and Mitophagy During Aging
Molecular Cell. 2019. Vol. 73, num. 4, p. 775 – 787.e10. DOI : 10.1016/j.molcel.2018.11.034.Altered interplay between endoplasmic reticulum and mitochondria in Charcot-Marie-Tooth type 2A neuropathy
Proceedings Of The National Academy Of Sciences Of The United States Of America. 2019. Vol. 116, num. 6, p. 2328 – 2337. DOI : 10.1073/pnas.1810932116.Compositions and methods for delivering nucleic acids to cochlear and vestibular cells
JP7430652; JP2024012440; EP3762040; JP2021519609; CN112423791; EP3762040; US2020392516; CA3093490; WO2019173367.
2019.A viral vector based in vivo model of tauopathy to explore therapeutic strategies against tau pathology
Lausanne, EPFL, 2019.Application of anti-amyloid beta immunization using encapsulated cell technology in mouse models of Alzheimer’s disease
Lausanne, EPFL, 2019.2018
An miRNA-based gene therapy approach to target mutated SOD1 in key cell types in amyotrophic lateral sclerosis (ALS)
2018. Conference on Changing the Face of Modern Medicine – Stem Cell and Gene Therapy, Lausanne, SWITZERLAND, Oct 16-19, 2018. p. A80 – A80.Pathogenic commonalities between spinal muscular atrophy and amyotrophic lateral sclerosis: Converging roads to therapeutic development
European Journal Of Medical Genetics. 2018. Vol. 61, num. 11, p. 685 – 698. DOI : 10.1016/j.ejmg.2017.12.001.Cas9/gRNAs Selective Targeting of the Beethoven Tmc-1 Mutant Allele for Treating Progressive Hearing Loss by AAV-Based Delivery
2018. 21st Annual Meeting of the American-Society-of-Gene-and-Cell-Therapy (ASGCT), Chicago, IL, May 16-19, 2018. p. 138 – 139.Endoplasmic reticulum and mitochondria in diseases of motor and sensory neurons: a broken relationship?
Cell Death & Disease. 2018. Vol. 9, p. 333. DOI : 10.1038/s41419-017-0125-1.PM20D1 is a quantitative trait locus associated with Alzheimer’s disease
Nature Medicine. 2018. Vol. 24, num. 5, p. 598 – 603. DOI : 10.1038/s41591-018-0013-y.G2019S LRRK2 enhances the neuronal transmission of tau in the mouse brain
2018. p. 120 – 134. DOI : 10.1093/hmg/ddx389.Exogenous LRRK2(G2019S) induces parkinsonian-like pathology in a nonhuman primate
JCI INSIGHT. 2018. Vol. 3, num. 14, p. e98202. DOI : 10.1172/jci.insight.98202.Encapsulation device
JP6417023; JP2018083819.
2018.Cortico–reticulo–spinal circuit reorganization enables functional recovery after severe spinal cord contusion
Nature Neuroscience. 2018. Vol. 21, num. 4, p. 576 – 588. DOI : 10.1038/s41593-018-0093-5.PFN2 and GAMT as common molecular determinants of axonal Charcot-Marie-Tooth disease
JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY. 2018. Vol. 89, num. 8, p. 870 – 878. DOI : 10.1136/jnnp-2017-317562.2017
In vivo neurochemical measurements in cerebral tissues using a droplet-based monitoring system
Nature Communications. 2017. Vol. 8, num. 1, p. 1239. DOI : 10.1038/s41467-017-01419-1.Bicistronic aav vector for rna interference in als
US11872290; HUE057809; EP4008789; ES2896181; SI3325632; PL3325632; LT3325632; PT3325632; DK3325632; EP3325632; US2021052744; US10835621; US2018228919; EP3325632; WO2017013252; EP3121284.
2017.