Results 31 to 40 of about 70,372 (263)
Adeno-associated virus: fit to serve [PDF]
Current Opinion in Virology, 2014 Adeno-associated virus (AAV) is a helper-dependent parvovirus which has not been linked with human disease. This aspect, in combination with its broad cell and tissue tropism, and limited viral host response has made it an attractive vector system for gene therapy.
Eric Zinn, Luk H Vandenberghe
openaire +2 more sources
Annals of Clinical and Translational Neurology, EarlyView.ABSTRACT Friedreich Ataxia (FRDA) is a neurodegenerative disorder of children and young adults associated with cardiomyopathy and other systemic complications. We report a 10‐year‐old girl who presented simultaneously with Acute Myelogenous Leukemia and FRDA who was successfully treated for her leukemia with allogeneic hematopoietic stem cell ...
Alexandra Gitman +5 more
wiley +1 more source
Advanced Healthcare Materials, EarlyView.Engineered red blood cell‐derived extracellular vesicles (eRBCEVs) are synthesized via controlled microfluidic assembly from native RBC lipids, enabling tunable encapsulation of proteins, nucleic acids, nanoparticles, and viral vectors. The platform demonstrates reproducible nanoscale architecture, preserved membrane composition, and functional cargo ...
Chiranth K. Nagaraj +23 more
wiley +1 more source
Adeno-Associated Virus VP1u Exhibits Protease Activity
Viruses, 2019 Adeno-associated viruses (AAVs) are being developed for gene delivery applications, with more than 100 ongoing clinical trials aimed at the treatment of monogenic diseases.
Justin J. Kurian +7 more
doaj +1 more source
Hydrogel Microtube Drug Carrier for Catheter‐Based Intravascular Therapy
Advanced Healthcare Materials, EarlyView.This paper proposes hydrogel microtube carriers composed of barium alginate gel, enabling long‐term vascular retention without interrupting blood flow. Intravascular treatments have long been attracting attention for therapeutic efficacy, yet practical delivery methods remained unestablished.
Shota Sato +8 more
wiley +1 more source
Journal of Extracellular Vesicles, 2023 Adeno‐associated virus (AAV) vector has shown multiple clinical breakthroughs, but its clinical implementation in inhaled gene therapy remains elusive due to difficulty in transducing lung airway cells.
Gijung Kwak +13 more
doaj +1 more source
Encoded Cell‐Material Interactions to Reroute Cytokine Signaling for Regenerative Medicine
Advanced Healthcare Materials, EarlyView.We present native MATRIX (Material Activated To Regulate Inducible gene eXpression), a co‐engineered material‐cell platform. Surfaces functionalized with antibodies for endogenous soluble ligands (e.g., IL‐1β or IL‐6) capture and immobilize ligands for presentation to a cognate, engineered synthetic Notch receptor, releasing a transcription factor (TF)
Zachary M. Eidman +7 more
wiley +1 more source
Pharmacology of Recombinant Adeno-associated Virus Production
Molecular Therapy: Methods & Clinical Development, 2018 Recombinant adeno-associated viral (rAAV) vectors have been used in more than 150 clinical trials with a good safety profile and significant clinical benefit in many genetic diseases.
Magalie Penaud-Budloo +3 more
doaj +1 more source
Nanomaterial Strategies for Pulmonary Delivery of Immunotherapeutics in Lung Cancer Treatment
Advanced Healthcare Materials, EarlyView.Inhalable immunotherapeutic nanomedicines enable organ‐selective immune modulation by overcoming pulmonary delivery barriers and concentrating therapy within lung tumors. This Review defines how nanomaterial properties govern airway deposition, retention, cellular partitioning, and immune activation across vaccines, checkpoint blockade, STING agonists,
Han Zhang, Wei Tang
wiley +1 more source
Advanced Healthcare Materials, EarlyView.A bicistronic rAAV8 vector encoding Aflibercept (Afb) and COMP‐cAng1 simultaneously coordinates VEGF clearance and Tie2‐mediated vessel maturation. In a 3D angiogenesis‐on‐a‐chip model, rAAV8‐Afb/cAng1 effectively reverses pathological barrier breakdown under clinical disease‐mimicking challenges.
Bong‐Kyu Kim +9 more
wiley +1 more source