[HTML][HTML] Biological Barriers for Drug Delivery and Development of Innovative Therapeutic Approaches in HIV, Pancreatic Cancer, and Hemophilia A/B
E Basar, H Mead, B Shum, I Rauter, C Ay… - Pharmaceutics, 2024 - mdpi.com
Biological barriers remain a major obstacle for the development of innovative therapeutics.
Depending on a disease's pathophysiology, the involved tissues, cell populations, and …
Depending on a disease's pathophysiology, the involved tissues, cell populations, and …
[HTML][HTML] Identification, Design, and Application of Noncoding Cis-Regulatory Elements
L Xu, Y Liu - Biomolecules, 2024 - mdpi.com
Cis-regulatory elements (CREs) play a pivotal role in orchestrating interactions with trans-
regulatory factors such as transcription factors, RNA-binding proteins, and noncoding RNAs …
regulatory factors such as transcription factors, RNA-binding proteins, and noncoding RNAs …
AAV-mediated co-expression of an immunogenic transgene plus PD-L1 enables sustained expression through immunological evasion
TB McMurphy, A Park, PJ Heizer, C Bottenfield… - Scientific Reports, 2024 - nature.com
Adeno-associated virus (AAV) vectors can mediate long-term expression of immunogenic
transgenes in vivo through transduction of tolerogenic cells in the liver. Tissue-targeted AAV …
transgenes in vivo through transduction of tolerogenic cells in the liver. Tissue-targeted AAV …
rAAV expressing a COBRA-designed influenza hemagglutinin generates a protective and durable adaptive immune response with a single dose
KB Wiggins, SM Winston, IL Reeves, J Gaevert… - Journal of …, 2024 - journals.asm.org
Influenza remains a worldwide public health threat. Although seasonal influenza vaccines
are currently the best means of preventing severe disease, the standard-of-care vaccines …
are currently the best means of preventing severe disease, the standard-of-care vaccines …
Coarse-Grained Simulations of Adeno-Associated Virus and Its Receptor Reveal Influences on Membrane Lipid Organization and Curvature
N Pipatpadungsin, K Chao… - The Journal of Physical …, 2024 - ACS Publications
Adeno-associated virus (AAV) is a well-known gene delivery tool with a wide range of
applications, including as a vector for gene therapies. However, the molecular mechanism of …
applications, including as a vector for gene therapies. However, the molecular mechanism of …
A humanized mouse model for adeno-associated viral gene therapy
Clinical translation of AAV-mediated gene therapy requires preclinical development across
different experimental models, often confounded by variable transduction efficiency. Here …
different experimental models, often confounded by variable transduction efficiency. Here …
CRISPR targeting of mmu-miR-21a through a single adeno-associated virus vector prolongs survival of glioblastoma-bearing mice
L Nieland, AB Vrijmoet, IW Jetten, D Rufino-Ramos… - Molecular Therapy, 2025 - cell.com
Glioblastoma (GB), the most aggressive tumor of the central nervous system (CNS), has
poor patient outcomes with limited effective treatments available. MicroRNA-21 (miR-21 (a)) …
poor patient outcomes with limited effective treatments available. MicroRNA-21 (miR-21 (a)) …
αFAP-specific nanobodies mediate a highly precise retargeting of modified AAV2 capsids thereby enabling specific transduction of tumor tissues
O Olarewaju, F Held, P Curtis, CH Kenny… - … Therapy Methods & …, 2024 - cell.com
Due to the refractiveness of tumor tissues to adeno-associated virus (AAV) transduction,
AAV vectors are poorly explored for cancer therapy delivery. Here, we aimed to engineer …
AAV vectors are poorly explored for cancer therapy delivery. Here, we aimed to engineer …
AAV-DJ is superior to AAV9 for targeting brain and spinal cord, and de-targeting liver across multiple delivery routes in mice
Highly efficient adeno associated viruses (AAVs) targeting the central nervous system (CNS)
are needed to deliver safe and effective therapies for inherited neurological disorders. The …
are needed to deliver safe and effective therapies for inherited neurological disorders. The …
RNA programmable cell targeting and manipulation with CellREADR
X Yang, K Woldemichael, J Hover, S Zhao, X Guo… - bioRxiv, 2024 - biorxiv.org
Methods that provide specific, easy, and scalable experimental access to animal cell types
and cell states will have broad applications in biology and medicine. CellREADR-Cell …
and cell states will have broad applications in biology and medicine. CellREADR-Cell …