[HTML][HTML] Emerging issues in AAV-mediated in vivo gene therapy

P Colella, G Ronzitti, F Mingozzi - Molecular Therapy-Methods & Clinical …, 2018 - cell.com
In recent years, the number of clinical trials in which adeno-associated virus (AAV) vectors
have been used for in vivo gene transfer has steadily increased. The excellent safety profile …

Bilirubin-induced neurologic damage—mechanisms and management approaches

JF Watchko, C Tiribelli - New England Journal of Medicine, 2013 - Mass Medical Soc
Bilirubin-Induced Neurologic Damage — Mechanisms and Management Approaches | New
England Journal of Medicine Skip to main content The New England Journal of Medicine …

Inherited disorders of bilirubin clearance

N Memon, BI Weinberger, T Hegyi, LM Aleksunes - Pediatric research, 2016 - nature.com
Inherited disorders of hyperbilirubinemia may be caused by increased bilirubin production
or decreased bilirubin clearance. Reduced hepatic bilirubin clearance can be due to …

Inherited disorders of bilirubin transport and conjugation: new insights into molecular mechanisms and consequences

S Erlinger, IM Arias, D Dhumeaux - Gastroenterology, 2014 - Elsevier
Inherited disorders of bilirubin metabolism might reduce bilirubin uptake by hepatocytes,
bilirubin conjugation, or secretion of bilirubin into bile. Reductions in uptake could increase …

Cardioprotective factors against myocardial infarction selected in vivo from an AAV secretome library

G Ruozi, F Bortolotti, A Mura, M Tomczyk… - Science translational …, 2022 - science.org
Therapies for patients with myocardial infarction and heart failure are urgently needed, in
light of the breadth of these conditions and lack of curative treatments. To systematically …

Bilirubin and beyond: a review of lipid status in Gilbert's syndrome and its relevance to cardiovascular disease protection

AC Bulmer, HJ Verkade, KH Wagner - Progress in lipid research, 2013 - Elsevier
Gilbert's syndrome (GS) is characterized by a benign, mildly elevated bilirubin concentration
in the blood. Recent reports show clear protection from cardiovascular disease in this …

Magnetic resonance imaging of bilirubin encephalopathy: current limitations and future promise

JL Wisnowski, A Panigrahy, MJ Painter… - Seminars in …, 2014 - Elsevier
Infants with chronic bilirubin encephalopathy often demonstrate abnormal bilateral,
symmetric, high-signal intensity on T2-weighted magnetic resonance imaging of the globus …

Promoterless gene targeting without nucleases rescues lethality of a Crigler‐Najjar syndrome mouse model

F Porro, G Bortolussi, A Barzel… - EMBO molecular …, 2017 - embopress.org
Crigler‐Najjar syndrome type I (CNSI) is a rare monogenic disease characterized by severe
neonatal unconjugated hyperbilirubinemia with a lifelong risk of neurological damage and …

Preclinical development of an AAV8-hUGT1A1 vector for the treatment of Crigler-Najjar syndrome

F Collaud, G Bortolussi, L Guianvarc'h… - … Therapy-Methods & …, 2019 - cell.com
Adeno-associated viruses (AAVs) are among the most efficient vectors for liver gene
therapy. Results obtained in the first hemophilia clinical trials demonstrated the long-term …

Bilirubin‐induced oxidative stress leads to DNA damage in the cerebellum of hyperbilirubinemic neonatal mice and activates DNA double‐strand break repair …

V Rawat, G Bortolussi, S Gazzin… - … medicine and cellular …, 2018 - Wiley Online Library
Unconjugated bilirubin is considered a potent antioxidant when present at moderate levels.
However, at high concentrations, it produces severe neurological damage and death …