Alberto Auricchio

Federico II University Hospital — Italy

Biochemistry, Genetics and Molecular Biology · Life Sciences

64h-index15.2kcitations244works6.72yr avg

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Research Topics

Virus-based gene therapy research(111), Retinal Development and Disorders(97), CRISPR and Genetic Engineering(48), Lysosomal Storage Disorders Research(22), Retinal Diseases and Treatments(21)

Publications238 total

Addressing Challenges in Developing Treatments for Inherited Retinal Diseases: Recommendations From the Third Monaciano Symposium

3 cited

Translational Vision Science & Technology·2025· 3 citedOpen Access

Retinal gene therapy for Stargardt disease with dual AAV intein vectors is both safe and effective in large animal models

9 cited

Science Advances·2025· 9 citedOpen Access

Multi-year enzyme expression in patients with mucopolysaccharidosis type VI after liver-directed gene therapy

7 cited

Med·2024· 7 citedOpen Access

Combined intraocular and intravenous gene delivery for therapy of gyrate atrophy of the choroid and retina

2 cited

Molecular Therapy·2024· 2 cited

Introduction to ESGCT 2024 Special Issue

Human Gene Therapy·2024

Safe and effective liver-directed AAV-mediated homology-independent targeted integration in mouse models of inherited diseases

15 cited

Cell Reports Medicine·2024· 15 citedOpen Access

Multicentric Longitudinal Prospective Study in a European Cohort of <i>MYO7A</i> Patients: Disease Course and Implications for Gene Therapy

6 cited

Investigative Ophthalmology & Visual Science·2024· 6 citedOpen Access

CoCas9 is a compact nuclease from the human microbiome for efficient and precise genome editing

16 cited

Nature Communications·2024· 16 citedOpen Access

Advances and Challenges in the Development of Gene Therapy Medicinal Products for Rare Diseases

15 cited

Human Gene Therapy·2023· 15 cited

Efficacy, pharmacokinetics, and safety in the mouse and primate retina of dual AAV vectors for Usher syndrome type 1B

24 cited

Molecular Therapy — Methods & Clinical Development·2023· 24 citedOpen Access

Liver‐directed gene therapy for ornithine aminotransferase deficiency

17 cited

EMBO Molecular Medicine·2023· 17 citedOpen Access

<scp>miR</scp> ‐181a/b downregulation: a mutation‐independent therapeutic approach for inherited retinal diseases

25 cited

EMBO Molecular Medicine·2022· 25 citedOpen Access

Full-length ATP7B reconstituted through protein trans-splicing corrects Wilson disease in mice

28 cited

Molecular Therapy — Methods & Clinical Development·2022· 28 citedOpen Access

Liver-Directed Adeno-Associated Virus–Mediated Gene Therapy for Mucopolysaccharidosis Type VI

26 cited

NEJM Evidence·2022· 26 citedOpen Access

Liver gene therapy with intein‐mediated F8 <i>trans</i> ‐splicing corrects mouse haemophilia A

13 cited

EMBO Molecular Medicine·2022· 13 citedOpen Access

Therapeutic homology-independent targeted integration in retina and liver

42 cited

Nature Communications·2022· 42 citedOpen Access

Inclusion of a degron reduces levels of undesired inteins after AAV-mediated protein trans-splicing in the retina

23 cited

Molecular Therapy — Methods & Clinical Development·2021· 23 citedOpen Access

The European Society of Gene and Cell Therapy: A Nearly 30-Year Endeavor to Make Gene Therapy a Clinical Reality

1 cited

Human Gene Therapy·2021· 1 cited

Zenodo (CERN European Organization for Nuclear Research)·2021Open Access

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Frequent Co-authors

Francesca Simonelli(14), Albert M. Maguire(12), Settimio Rossi(11), Jean Bennett(11), James M. Wilson(10), Enrico Maria Surace(9), J. Fraser Wright(8), Sandro Banfi(7), Kathleen Marshall(7), Francesco Testa(7), Markus Hildinger(7), Eric A. Pierce(6), Junwei Sun(6), Katherine A. High(6), Erin E. O’Connor(6), Andrea Ballabio(6), Ivana Trapani(6), Federico Mingozzi(5), Jeannette Bennicelli(5), Kenneth S. Shindler(5)