Marco De Giorgi

Nanomedicine beyond carriers — devices, cells & living therapeutics

Ryan Williams, Ravit Yakobi Arancibia, Weiwei Gao, Rohan Palanki et autres

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Nanomedicine beyond carriers — devices, cells & living therapeutics

Alessandro Grattoni, Marco Maria Paci, Noah Arnold, Santosh Aryal et autres

Nanomedicine has progressed far beyond its early role as an experimental drug-carrier toolbox and today stands as a clinically validated enabling technology. Liposomal formulations and albumin-bound nanoparticles have transformed cancer therapy, while lipid nanoparticle (LNP) platforms accelerated the rapid development and global …

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Cytosine base editing of LPA in transgenic mice averts large deletions

Marcel Chuecos, So Hyun Park, Madhvi Bhakta, Usosa Too-Chiobi et autres

Lipoprotein(a) (Lp(a)) is a genetically determined causal risk factor for cardiovascular disease, with approximately 20% of the population exhibiting elevated levels. While there are promising drugs in development, there are currently no approved therapies specifically designed to lower Lp(a) levels. For high-risk …

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Repair Drive improves gene editing in the liver

Marco De Giorgi, So Hyun Park, Gang Bao, William R. Lagor

The liver is a vital metabolic hub for the entire body, performing essential tasks in amino acid and glucose metabolism, drug detoxification, protein secretion, thrombosis, fibrinolysis, and lipid homeostasis. There are at least 700 monogenic diseases that could be treated with gene …

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In vivo expansion of gene-targeted hepatocytes through transient inhibition of an essential gene

Marco De Giorgi, So Hyun Park, Adam Castoreno, Mingming Cao et autres

Homology-directed repair (HDR)–based genome editing is an approach that could permanently correct a broad range of genetic diseases. However, its utility is limited by inefficient and imprecise DNA repair mechanisms in terminally differentiated tissues. Here, we tested Repair Drive, a platform technology …

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A comprehensive atlas of AAV tropism in the mouse

Christopher J. Walkey, Kathy J. Snow, Jote Bulcha, Aaron R. Cox et autres

Gene therapy with adeno-associated virus (AAV) vectors requires knowledge of their tropism within the body. Here we analyze the tropism of 10 naturally occurring AAV serotypes (AAV3B, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAVrh8, AAVrh10, and AAVrh74) following systemic delivery into male …

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N-Acetyl-leucine in progressive CACNA1A ataxia: A case series

Κyriakos Martakis, Marco De Giorgi, Maria Spanou, Bernd A. Neubauer et autres

BACKGROUND: CACNA1A-related disorders are rare and progressive; to date, there is no approved treatment. Trials with N-acetyl-leucine (NAL) demonstrated efficacy in disorders featuring ataxia, cognitive impairment, and epilepsy. Accordingly, we hypothesized that NAL may be effective in CACNA1A-associated disorders. CASES: Four patients …

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Ectonucleotidases

Raíssa Leite-Aguiar, Marco De Giorgi, Luiz Eduardo Baggio Savio, Simon C. Robson

Latest results from precision measurements at the NA62 experiment

Petre Boboc, A. Akmete, R. Aliberti, F. Ambrosino et autres

Then NA62 experiment at CERN collected the world's largest dataset of charged Kaons, the main goal being the measurement of the $\mathcal{B}(K^+ \rightarrow \pi^+ \nu \bar{\nu})$. New results from the analyses of rare kaon and pion decays using data samples collected in …

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N-Acetyl-DL-Leucine Improves Ataxia in Patients with CACNA1A-Related Disorders

Κyriakos Martakis, Marco De Giorgi, Thea Beatrice Abele, Maria Spanou et autres

Background/Purpose: CACNA1A-related disorders are rare, progressive, and prematurely fatal. They can manifest with a variety of symptoms, such as ataxia, epilepsy, central hypotonia, developmental delay or intellectual disability. To date, there is no approved specific treatment. N -acetyl-DL-leucine (NAL) is an orally …

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The Role of Machine Learning in the Most Common Hematological Malignancies: A Narrative Review

Teresa Perillo, Marco De Giorgi, Claudia Giorgio, Carmine Frasca et autres

Background: Hematologic malignancies are a group of heterogeneous neoplasms which originate from hematopoietic cells. The most common among them are leukemia, lymphoma, and multiple myeloma. Machine learning (ML) is a subfield of artificial intelligence that enables the analysis of large amounts of …

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A Comprehensive Atlas of AAV Tropism in the Mouse

Christopher J. Walkey, Kathy J. Snow, Jote Bulcha, Aaron R. Cox et autres

Gene therapy with Adeno-Associated Viral (AAV) vectors requires knowledge of their tropism within the body. Here we analyze the tropism of ten naturally occurring AAV serotypes (AAV3B, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAVrh8, AAVrh10 and AAVrh74) following systemic delivery into male …

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