Vania Broccoli

Vita-Salute San Raffaele University — Italy

Biochemistry, Genetics and Molecular Biology · Life Sciences

65h-index15.6kcitations198works5.02yr avg
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Research Topics

Genetics and Neurodevelopmental Disorders(38), Pluripotent Stem Cells Research(36), CRISPR and Genetic Engineering(29), Neurogenesis and neuroplasticity mechanisms(27), Developmental Biology and Gene Regulation(21)

Publications198 total

A fully human pluripotent stem cell-derived blood-brain barrier model for clinically relevant disease modeling and selection of neurotropic adeno-associated viruses
2025Open Access
Targeted and efficient AAV therapy for neuroblastoma via direct capsid-antibody coupling
2025
Unraveling the role of GBA1 genotype in axial signs response to subthalamic deep brain stimulation
npj Parkinson s Disease·2025Open Access
2024 IRSF Rett Syndrome Scientific Meeting June 18-19, 2024, Westminster, Colorado, USA
Translational Science of Rare Diseases·2024Open Access
A WFS1 variant disrupting acceptor splice site uncovers the impact of alternative splicing on beta cell apoptosis in a patient with Wolfram syndrome
Diabetologia·2024· 5 citedOpen Access
Acid-sensing ion channel 3 is a new potential therapeutic target for the control of glioblastoma cancer stem cells growth
Scientific Reports·2024· 7 citedOpen Access
Recreating Leber’s Hereditary Optic Neuropathy in iPS derived Neurons: Shedding Light on Pathogenic Mechanisms to Combat Blindness
Biochimica et Biophysica Acta (BBA) - Bioenergetics·2024
Microglia-specific <i>IL-10</i> gene delivery inhibits neuroinflammation and neurodegeneration in a mouse model of Parkinson’s disease
Science Translational Medicine·2024· 34 cited
Selective homing of CAR-CIK cells to the bone marrow niche enhances control of the acute myeloid leukemia burden
UNC Libraries·2024Open Access
New AAV9 engineered variants with enhanced neurotropism and reduced liver off-targeting in mice and marmosets
iScience·2024· 12 citedOpen Access
Shedding a Light on Dark Genes: A Comparative Expression Study of PRR12 Orthologues during Zebrafish Development
Genes·2024· 1 citedOpen Access
Generation of iPSCs from identical twin, one affected by LHON and one unaffected, both carrying a combination of two mitochondrial variants: m.14484 T&gt;C and m.10680G&gt;A
Stem Cell Research·2024· 1 citedOpen Access
A <i>WFS1</i> variant disrupting acceptor splice site uncovers the impact of alternative splicing on β cell apoptosis in a patient with Wolfram syndrome
2023· 1 citedOpen Access
Reply: Spatial and temporal manipulation of the<i>Scn1a</i>gene affect adult brain function
Brain·2023· 1 citedOpen Access
Enhanced production of mesencephalic dopaminergic neurons from lineage-restricted human undifferentiated stem cells
Nature Communications·2023· 10 citedOpen Access
Temporal manipulation of the <i>Scn1a</i> gene reveals its essential role in adult brain function
Brain·2023· 10 citedOpen Access
Genome-wide screening in pluripotent cells identifies Mtf1 as a suppressor of mutant huntingtin toxicity
Nature Communications·2023· 9 citedOpen Access
New AAV9 engineered variants with enhanced neurotropism and reduced liver off-targeting in mice and marmosets
2023· 1 citedOpen Access
Balanced SET levels favor the correct enhancer repertoire during cell fate acquisition
Nature Communications·2023· 15 citedOpen Access
SETD5 haploinsufficiency affects mitochondrial compartment in neural cells
Molecular Autism·2023· 4 citedOpen Access
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Frequent Co-authors

Alessandro Sessa(10), Luca Massimino(9), Serena Giannelli(9), Gaia Colasante(8), Alicia Rubio(6), Stefano Taverna(5), Fabio Benfenati(5), Massimiliano Caiazzo(4), Edoardo Boncinelli(4), Andrea Ballabio(4), Simone Bido(4), Gabriele Lignani(4), Massimo Gulisano(4), Maria Teresa Dell’Anno(3), Elena Dvoretskova(3), Damiana Leo(3), Raul R. Gainetdinov(3), Alexander Dityatev(3), Sara Ricciardi(3), Wolfgang Wurst(3)