Results 141 to 150 of about 47,774 (288)
Integrin Targeted Delivery of Gene Therapeutics
Theranostics, 2011 Integrins have become key targets for molecular imaging and for selective delivery of anti-cancer agents. Here we review recent work concerning the targeted delivery of antisense and siRNA oligonucleotides via integrins. A variety of approaches have been
Rudy L Juliano, Xin Ming, Osamu Nakagawa, Rongzuo Xu, Hoon Yoo
doaj
International Journal of Cancer, EarlyView.Clinical trials of chimeric antigen receptor T‐cell (CAR‐T) therapies in glioblastoma have shown limited clinical benefits. Whether this may be explained by the basal quality of CAR‐T products, which are currently generated using patient, autologous T‐cells, has been little explored.
Sabra K. Salim +22 more
wiley +1 more source
iNew Medicine, EarlyView.A tFNA‐based siRNA delivery system targeting IGFBP7 effectively silences IGFBP7 expression and attenuates renal dysfunction, tubular injury, and cell death in both septic (CLP) and ischemic (I/R) murine models of acute kidney injury. ABSTRACT Acute kidney injury (AKI) is a common and serious clinical condition, yet effective targeted therapies remain ...
Shao‐qi Wu +11 more
wiley +1 more source
Splicing therapeutics in SMN2 and APOB
, 2009 Splicing therapeutics are defined as the deliberate modification of RNA splicing to achieve therapeutic goals. Various techniques for splicing therapeutics have been described, and most of these involve the use of antisense oligonucleotide-based ...
Krainer, AR +3 more
core
The regulation of stem cell fate and its application in neural regeneration
Interdisciplinary Medicine, EarlyView.Regulating stem cell fate is crucial for neural regeneration. This review summarizes key physical, biological, and chemical strategies and their applications in repairing nerve injuries, providing new insights for regenerative medicine. Abstract Regulating the fate of stem cells (SCs) is a key technical problem in the field of regenerative medicine and
Yuexin He +3 more
wiley +1 more source
Journal of the Chinese Chemical Society, EarlyView.Model for how α‐syn modulates the positioning of endolysosomes in melanoma cells. (a) α‐syn tethers endolysosomes to the plasma membrane, a last step in anterograde transport. (b) Loss of α‐syn expression causes the loss of the tethering function, which leads to perinuclear vesicle clustering. Reproduced from the open access article.
Stephan N. Witt
wiley +1 more source
Muscle &Nerve, EarlyView.ABSTRACT Aims We report 5‐year results from a phase 1/2a study of delandistrogene moxeparvovec, a recombinant adeno‐associated virus serotype rh74 vector‐based gene therapy for Duchenne muscular dystrophy (DMD), with post hoc analyses contextualizing functional outcomes. Methods Four ambulatory patients with DMD (≥ 4–< 8 years at enrollment) entered an
Jerry R. Mendell +10 more
wiley +1 more source
Reduction-sensitive polymers and bioconjugates for biomedical applications
, 2009 Reduction-sensitive biodegradable polymers and conjugates have emerged as a fascinating class of biomedical materials that can be elegantly applied for intracellular triggered gene and drug delivery.
Meng, F.H. +4 more
core
Nanodiamonds (NDs) are considered promising delivery platforms, but inaccurate and uncontrolled release of drugs at target sites is the biggest challenge of NDs in precision medicine.
Ling Sum Liu (3850741) +7 more
core +1 more source
Muscle &Nerve, EarlyView.ABSTRACT Introduction/Aims Tofersen is a gene‐targeted therapy for individuals with superoxide dismutase 1 (SOD1) (+) amyotrophic lateral sclerosis (ALS). Prior to U.S. Food and Drug Administration (FDA) approval, tofersen was made available through expanded access protocol.
Alison Wheeler +30 more
wiley +1 more source