Mariangela Lupo

Telethon Institute Of Genetics And Medicine — Italy

Biochemistry, Genetics and Molecular Biology · Life Sciences

5h-index271citations14works8.32yr avg
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Research Topics

Retinal Development and Disorders(8), CRISPR and Genetic Engineering(5), Photoreceptor and optogenetics research(4), Virus-based gene therapy research(3), Plant Molecular Biology Research(2)

Publications14 total

Retinal gene therapy for Stargardt disease with dual AAV intein vectors is both safe and effective in large animal models
Science Advances·2025· 9 citedOpen Access
A DNA base-specific sequence interposed between CRX and NRL contributes to RHODOPSIN expression
Scientific Reports·2024Open Access
CoCas9 is a compact nuclease from the human microbiome for efficient and precise genome editing
Nature Communications·2024· 16 citedOpen Access
A DNA base-specific sequence interposed between CRX and NRL contributes to RHODOPSIN expression.
2023Open Access
Inclusion of a degron reduces levels of undesired inteins after AAV-mediated protein trans-splicing in the retina
Molecular Therapy — Methods & Clinical Development·2021· 23 citedOpen Access
Intein-mediated protein trans-splicing expands adeno-associated virus transfer capacity in the retina
Science Translational Medicine·2019· 177 citedOpen Access
Modulating transcription factor spatial pattern to generate gene-targeted therapeutics
Investigative Ophthalmology & Visual Science·2018Open Access
RHODOPSIN transcriptional silencing & replacement for the treatment of autosomal dominant Retinitis Pigmentosa
Investigative Ophthalmology & Visual Science·2018Open Access
736. Silencing Substituting the RHODODPSIN Gene to Treat Blinding Disorders
Molecular Therapy·2016Open Access
320. Transcriptional Silencing via Synthetic DNA Binding Protein Lacking Canonical Repressor Domains as a Potent Tool to Generate Therapeutics
Molecular Therapy·2016Open Access
Rhodopsin targeted transcriptional silencing by DNA-binding
eLife·2016· 46 citedOpen Access
Author response: Rhodopsin targeted transcriptional silencing by DNA-binding
2016Open Access
484. Preclinical Proof of Concept of Transcriptional Silencing and Replacement Strategy for Treatment of Retinitis Pigmentosa Due To RHODOPSIN Mutations
Molecular Therapy·2015Open Access
CHROMOSOME MODIFICATIONS OF HUMAN LYMPHOCYTES AFTER STRESS.
OSTI OAI (U.S. Department of Energy Office of Scientific and Technical Information)·1966

Frequent Co-authors

Enrico Maria Surace(12), Elena Marrocco(11), Francesca Simonelli(10), Carlo Gesualdo(9), Settimio Rossi(9), Salvatore Botta(9), Martina Sofia(9), Maria Laura Bacci(9), Nicola de Prisco(7), Mario Renda(7), Fabiola Curion(5), Alberto Auricchio(4), Ivana Trapani(3), Patrizia Tornabene(2), Renato Minopoli(2), Miriam Centrulo(2), Paola Tiberi(2), Carolina Iodice(2), Annamaria Carissimo(2), Rosa Maritato(2)