Results 91 to 100 of about 20,871,284 (295)

Neuroimaging Applications for the Delivery and Monitoring of Gene Therapy for Central Nervous System Diseases

Neurological disease due to single gene defects represents a targetable entity for adeno-associated virus (AAV) mediated gene therapy. The delivery of AAV-mediated gene therapy to the brain is challenging, owing to the presence of the blood-brain barrier.
Vardar, Zeynep, Gray-Edwards, Heather L, Daci, Rrita, Flotte, Terence R, Shazeeb, Mohammed Salman, Cataltepe, Oguz I, Vachha, Behroze   +6 more
core   +1 more source

Liver organoids: modelling complexity in homeostasis and disease

FEBS Letters, EarlyView.
Studying liver in vitro has been challenging because simple 2D cell cultures fail to capture liver's cellular and architectural complexity. To bridge this gap, scientists increasingly use organoids, 3D liver models which better mimic liver composition and function. This review examines recent advances in liver organoid complexity and realism, discusses
Anna M. Dowbaj, Meritxell Huch
wiley   +1 more source

Transferrin-targeting redox hyperbranched poly(amido amine)-functionalized graphene oxide for sensitized chemotherapy combined with gene therapy to nasopharyngeal carcinoma

Drug Delivery, 2019
A drug and gene co-delivery system with chemotherapeutic sensibilization was prepared and used for nasopharyngeal carcinoma therapy. For this purpose, the graphene oxide (GO) was conjugated with the redox hyperbranched poly(amido amine) (HPAA) and then ...
Tao Liu, Jingzhen Li, Xidong Wu, Siyi Zhang, Zhongming Lu, Guanxue Li, Junzheng Li, Shaohua Chen   +7 more
doaj   +1 more source

Targeting of Synthetic Gene Delivery Systems [PDF]

BioMed Research International, 2003
Safe, efficient, and specific delivery of therapeutic genes remains an important bottleneck for the development of gene therapy. Synthetic, nonviral systems have a unique pharmaceutical profile with potential advantages for certain applications. Targeting of the synthetic vector improves the specificity of gene medicines through a modulation of the ...
openaire   +2 more sources

Autophagy and mitophagy in pancreatic β‐cell homeostasis and their involvement in diabetes pathophysiology

FEBS Letters, EarlyView.
This review focuses on the role of autophagy and mitophagy in maintaining pancreatic β‐cell function and homeostasis. We discuss how genetic defects affecting these pathways contribute to the development of type 1, type 2, monogenic, and gestational diabetes. We further explore their potential as therapeutic targets. Created in BioRender.
Yunkyeong Lee, Mingming Tong, Anna L. Gloyn   +2 more
wiley   +1 more source

Developing a Novel Gene-Delivery Vector System Using the Recombinant Fusion Protein of Pseudomonas Exotoxin A and Hyperthermophilic Archaeal Histone HPhA.

PLoS ONE, 2015
Non-viral gene delivery system with many advantages has a great potential for the future of gene therapy. One inherent obstacle of such approach is the uptake by endocytosis into vesicular compartments.
Xin Deng, Guoli Zhang, Ling Zhang, Yan Feng, Zehong Li, GuangMou Wu, Yuhuan Yue, Gensong Li, Yu Cao, Ping Zhu   +9 more
doaj   +1 more source

The gene delivery system for rheumatoid synovium

Japanese Journal of Clinical Immunology, 2008
A gene therapy of synovial tissue is potentially a novel therapeutic method in rheumatoid arthritis (RA). The method induces expression of anti-arthritic molecules in target cells, and is also useful for the investigation of novel therapeutic targets in vitro.
NAKASATO, Makoto, NONOMURA, Yoshinori, MIYASAKA, Nobuyuki, KOHSAKA, Hitoshi   +3 more
openaire   +3 more sources

Redosing Adeno-Associated Virus Gene Therapy to the Central Nervous System

, 2022
Adeno-associated virus (AAV)-mediated gene therapies have provided promising treatments for numerous neurological disorders. Redosing of AAV to the central nervous system (CNS) is an attractive research area due to both the somewhat immunologically ...
Arjomandnejad, Motahareh, Gray-Edwards, Heather L, Sena-Esteves, Miguel, McElroy, Abigail, Keeler, Allison M   +4 more
core   +1 more source

Golgi enzymes are retrieved from the plasma membrane to the trans‐Golgi network

FEBS Letters, EarlyView.
Golgi enzymes are traditionally considered resident proteins retained within the Golgi apparatus. Here, we demonstrate that a subset transiently reaches the cell surface and is subsequently retrieved to the trans‐Golgi network via retrograde transport. Using a nanobody‐based toolkit, we uncover a dynamic trafficking cycle of several Golgi enzymes.
Dominik P. Buser, Tina Junne
wiley   +1 more source

Translophagy—A potential link between autophagy impairment and translational errors

FEBS Letters, EarlyView.
Neurodegenerative diseases are characterised by the accumulation of abnormal proteins and protein aggregates, but their origin often remains unknown. We propose that selective autophagy removes damaged protein‐making machinery, preventing errors during protein synthesis.
Mykola V. Korolchuk, Gailing Ma, Robin Antrobus, Diego Manni, Marco Trevisan‐Herraz, Mustafa Khwaja, Max Brown, Gavin D. Richardson, Masaaki Komatsu, Daniel Erskine, Viktor I. Korolchuk, Tetsushi Kataura   +11 more
wiley   +1 more source