Accès ouvert
2026
article
OpenAlex
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 …
us, ca
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2025
article
OpenAlex
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 …
us, ca
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Accès ouvert
2025
article
OpenAlex
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 …
us
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Accès ouvert
2024
preprint
OpenAlex
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 …
us, es
(code pays fourni par la source)
Accès ouvert
2023
preprint
OpenAlex
Marco De Giorgi, So Hyun Park, Adam Castoreno, Mingming Cao et autres
Abstract 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 …
us
(code pays fourni par la source)
Accès ouvert
2023
article
OpenAlex
Marcel Chuecos, William R. Lagor
The liver is the metabolic center of the body and an ideal target for gene therapy of inherited metabolic disorders (IMDs). Adeno-associated viral (AAV) vectors can deliver transgenes to the liver with high efficiency and specificity and a favorable safety profile. Recombinant …
us
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2023
conference-abstract
OpenAlex
Marcel Chuecos, Marco De Giorgi, Christopher J. Walkey, Bryan Nguyen et autres
The mevalonate pathway is an important metabolic pathway that provides cells with vital bioactive molecules. The pathway converts mevalonate into various sterol and nonsterol isoprenoids required for cellular maintenance and health, including, cholesterol, ubiquinione, dolichol, heme A, farnesyl, and gernaylgeranyl protein anchors. …
us
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Accès ouvert
2022
article
OpenAlex
Alexandria M. Doerfler, So Hyun Park, Julia M. Assini, Amer Youssef et autres
Lipoprotein(a) (Lp(a)) represents a unique subclass of circulating lipoprotein particles and consists of an apolipoprotein(a) (apo(a)) molecule covalently bound to apolipoprotein B-100. The metabolism of Lp(a) particles is distinct from that of low-density lipoprotein (LDL) cholesterol, and currently approved lipid-lowering drugs do …
us, ca
(code pays fourni par la source)
2021
conference-abstract
OpenAlex
Alexandria M. Doerfler, Jun Han, Li Tang, Marco De Giorgi et autres
While the intestine is the critical interface between cholesterol absorption and excretion, surprisingly little is known about the role of de novo cholesterol synthesis in this organ and how it affects whole body cholesterol homeostasis. The mevalonate pathway is most well-known for …
us, ca
(code pays fourni par la source)
Accès ouvert
2020
article
OpenAlex
Rebecca L. Wilson, Kathryn Owens, Emily K. Sumser, Matthew V. Fry et autres
us
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Accès ouvert
2018
article
OpenAlex
Maira Carrillo, Marcel Chuecos, Kushal Gandhi, Andrey A. Bednov et autres
Imaging of placental tissues is a difficult task, because of specific for this organ complex multicellular and 3D tissue structure. The tissue clearing systems (X-CLARITY) system is a valuable tool for the examining the expression of molecular pathways in whole tissues and …
us
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Accès ouvert
2017
article
OpenAlex
Kushal Gandhi, Cun Li, Nadezhda German, Cezary Skobowiat et autres
Maternal obesity in pregnancy has been linked to a spectrum of adverse developmental changes. Involvement of eCBs in obesity is well characterized. However, information regarding eCB physiology in obesity associated with pregnancy is sparse. This study evaluated fetomaternal hepatic, systemic, and placental …
us
(code pays fourni par la source)