Results 91 to 100 of about 144,727 (384)
Frontiers in GeneticsIntroduction: Transcriptional regulation is an important process wherein non-protein coding enhancer sequences play a key role in determining cell type identity and phenotypic diversity.
Ashley R. Brown +27 more
doaj +1 more source
Cortical AAV-CNTF gene therapy combined with intraspinal mesenchymal precursor cell transplantation promotes functional and morphological outcomes after spinal cord injury in adult rats [PDF]
, 2018 Ciliary neurotrophic factor (CNTF) promotes survival and enhances long-distance regeneration of injured axons in parts of the adult CNS. Here we tested whether CNTF gene therapy targeting corticospinal neurons (CSN) in motor-related regions of the ...
Akinpelu, Emmanuel A +14 more
core +3 more sources
bioRxiv, 2019 The engineered AAV-PHP.B family of adeno-associated virus efficiently delivers genes throughout the mouse central nervous system. To guide their application across disease models, and to inspire the development of translational gene therapy vectors ...
Qin Huang +10 more
semanticscholar +1 more source
Rescuing AAV gene transfer from neutralizing antibodies with an IgG-degrading enzyme
JCI Insight, 2020 Preexisting humoral immunity to recombinant adeno-associated virus (AAV) vectors restricts the treatable patient population and efficacy of human gene therapies.
Zachary C. Elmore +4 more
semanticscholar +1 more source
Lecithin Alleviates Memory Deficits and Muscle Attenuation in Chinese Older Adults and SAMP8 Mice
Advanced Science, EarlyView.This study opens a new avenue for safeguarding cognition and muscle health, averting disability in older age, and treating age‐related pathologies through lecithin supplementation. It serves as a promising nonpharmacological intervention for the crosstalk of muscle and cognition.
Xianyun Wang +16 more
wiley +1 more source
Targeting tauopathy with engineered tau-degrading intrabodies [PDF]
, 2019 BACKGROUND: The accumulation of pathological tau is the main component of neurofibrillary tangles and other tau aggregates in several neurodegenerative diseases, referred to as tauopathies.
Gallardo, Gilbert +7 more
core +1 more source
AAV capsid CD8+ T-cell epitopes are highly conserved across AAV serotypes
Molecular Therapy - Methods & Clinical Development, 2015 Adeno-associated virus (AAV) has become one of the most promising vectors in gene transfer in the last 10 years with successful translation to clinical trials in humans and even market approval for a first gene therapy product in Europe. Administration to humans, however, revealed that adaptive immune responses against the vector capsid can present an ...
Daniel J Hui +9 more
openaire +4 more sources
Self‐Adjusting Engineered Probiotic for Targeted Tumor Colonization and Local Therapeutics Delivery
Advanced Science, EarlyView.Zou et al. developed an engineered strain of Escherichia coli Nissle 1917 with precise targeting abilities for colonization and on‐demand payload release. The engineered probiotic survives and embolizes tumors only in the presence of more than 5 mM L‐lactate. Furthermore, the introduction of an α‐hemolysin circuit enhances its anti‐tumor effect.
Zhen‐Ping Zou +8 more
wiley +1 more source
Long-term Evaluation of AAV-CRISPR Genome Editing for Duchenne Muscular Dystrophy
Nature Network Boston, 2018 Duchenne muscular dystrophy (DMD) is a monogenic disorder and a candidate for therapeutic genome editing. There have been several recent reports of genome editing in preclinical models of Duchenne muscular dystrophy1–6, however, the long-term persistence
Christopher E. Nelson +11 more
semanticscholar +1 more source
Advanced Science, EarlyView.Current preclinical studies of AAV‐mediated gene therapy explore different strategies based on the characteristics of inner ear diseases. For genetic hearing loss, approaches include the replacement of a “good gene,” removal of a “bad gene,” or direct correction of mutations through base editing.
Fan Wu +7 more
wiley +1 more source