Results 31 to 40 of about 32,863 (259)
AAV genome modification for efficient AAV production
Heliyon, 2023 The adeno-associated virus (AAV) is one of the most potent vectors in gene therapy. The experimental profile of this vector shows its efficiency and accepted safety, which explains its increased usage by scientists for the research and treatment of a ...
Walaa Asaad +6 more
doaj +1 more source
BiomoleculesIn this study, we introduce electrospun polydioxanone (PDO) nonwoven fabrics as a platform for the delivery of adeno-associated virus (AAV) vectors for transduction and genome editing by adhering them to organ surfaces, including the heart.
Kotoko Furuno +2 more
doaj +1 more source
Molecular Therapy, 2003 One of the many hurdles that face gene therapists is the requirement to optimize gene delivery systems in such a way that both efficient and selective transgene production is achieved in vivo within a chosen cell or tissue. Exploiting native vector tropism can sometimes achieve this goal irrespective of additional modifications. For example, adenoviral
openaire +2 more sources
Immunogenicity and toxicity of AAV gene therapy
Frontiers in Immunology, 2022 Gene transfer using adeno-associated viral (AAV) vectors has made tremendous progress in the last decade and has achieved cures of debilitating diseases such as hemophilia A and B.
Hildegund C. J. Ertl
doaj +1 more source
The state of the art of adeno-associated virus-based vectors in gene therapy
Virology Journal, 2007 The adeno-associated virus (AAV) has rapidly gained popularity in gene therapy since the establishment of the first AAV2 infectious clone, in 1982, due to some of their distinguishing characteristics such as lack of pathogenicity, wide range of ...
Nardi Nance, Coura Renata
doaj +1 more source
Molecular Therapy: Methods & Clinical DevelopmentVectors based on adeno-associated viruses (AAVs) are promising therapeutic modalities used in gene therapy. Robust cell-based assays that demonstrate and quantify the potency of AAV vectors in expressing their transgene are needed for clinical ...
James Zengel +4 more
doaj +1 more source
Molecular Therapy: Methods & Clinical Development, 2020 Adeno-associated viral (AAV) vectors are attractive tools for central nervous system (CNS) gene therapy because some vectors can cross the blood-brain barrier (BBB), allowing them to be used as minimally invasive treatments.
Sophie N. Mathiesen +4 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
Molecular Therapy: Methods & Clinical Development, 2014 The muscular dystrophies are a group of devastating genetic disorders that affect both skeletal and cardiac muscle. An effective gene therapy for these diseases requires bodywide muscle delivery.
Chady H Hakim +6 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