Results 41 to 50 of about 647,332 (140)

Progress in the use of adeno-associated viral vectors for gene therapy [PDF]

open access: yes, 2004
The development of safe and efficient gene transfer vectors is crucial for the success of gene therapy trials. A viral vector system promising to meet these requirements is based on the apathogenic adeno-associated virus (AAV-2), a member of the ...
Braun-Falco, M., Buning, H., Hallek, M.
core   +1 more source

In vivo armed macrophages curb liver metastasis through tumor-reactive T-cell rejuvenation

open access: yesNature Communications
Despite recent progress in cancer treatment, liver metastases persist as an unmet clinical need. Here, we show that arming liver and tumor-associated macrophages in vivo to co-express tumor antigens (TAs), IFNα, and IL-12 unleashes robust anti-tumor ...
Marco Notaro   +20 more
doaj   +1 more source

Efficient Gene Targeting Mediated by Adeno-Associated Virus and DNA Double-Strand Breaks [PDF]

open access: yes, 2003
Gene targeting is the in situ manipulation of the sequence of an endogenous gene by the introduction of homologous exogenous DNA. Presently, the rate of gene targeting is too low for it to be broadly used in mammalian somatic cell genetics or to cure ...
Baltimore, David   +3 more
core  

Targeted tandem affinity purification of PSD‐95 recovers core postsynaptic complexes and schizophrenia susceptibility proteins

open access: yesMolecular Systems Biology, 2009
The molecular complexity of mammalian proteomes demands new methods for mapping the organization of multiprotein complexes. Here, we combine mouse genetics and proteomics to characterize synapse protein complexes and interaction networks.
Esperanza Fernández   +9 more
doaj   +1 more source

Novel Heterotypic Rox Sites for Combinatorial Dre Recombination Strategies

open access: yesG3: Genes, Genomes, Genetics, 2016
Site-specific recombinases (SSRs) such as Cre are widely used in gene targeting and genetic approaches for cell labeling and manipulation. They mediate DNA strand exchange between two DNA molecules at dedicated recognition sites. Precise understanding of
Katherine Chuang   +3 more
doaj   +1 more source

iPSC-Derived Intestinal Organoids from Cystic Fibrosis Patients Acquire CFTR Activity upon TALEN-Mediated Repair of the p.F508del Mutation

open access: yesMolecular Therapy: Methods & Clinical Development, 2020
Cystic fibrosis (CF) is the main genetic cause of death among the Caucasian population. The disease is characterized by abnormal fluid and electrolyte mobility across secretory epithelia.
Aarne Fleischer   +12 more
doaj   +1 more source

Artificial Intelligence and Precision Medicine: A New Frontier for the Treatment of Brain Tumors

open access: yesLife, 2022
Brain tumors are a widespread and serious neurological phenomenon that can be life- threatening. The computing field has allowed for the development of artificial intelligence (AI), which can mimic the neural network of the human brain.
Anil K. Philip   +4 more
doaj   +1 more source

Inhibition of Murine Cytomegalovirus Infection in Animals by RNase P-Associated External Guide Sequences. [PDF]

open access: yes, 2017
External guide sequence (EGS) RNAs are associated with ribonuclease P (RNase P), a tRNA processing enzyme, and represent promising agents for gene-targeting applications as they can direct RNase-P-mediated cleavage of a target mRNA.
Li, Wei   +9 more
core   +2 more sources

LPG2 Gene Duplication in Leishmania infantum: A Case for CRISPR-Cas9 Gene Editing

open access: yesFrontiers in Cellular and Infection Microbiology, 2020
On the surface of the Leishmania promastigote, phosphoglycans (PG) such as lipophosphoglycan (LPG), proteophosphoglycan (PPG), free phosphoglycan polymers (PGs), and acid phosphatases (sAP), are dominant and contribute to the invasion and survival of ...
Flávio Henrique Jesus-Santos   +9 more
doaj   +1 more source

Improvement of the LbCas12a-crRNA System for Efficient Gene Targeting in Tomato

open access: yesFrontiers in Plant Science, 2021
Plant gene targeting (GT) can be utilized to precisely replace up to several kilobases of a plant genome. Recent studies using the powerful clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated (Cas) nucleases ...
Tien Van Vu   +8 more
doaj   +1 more source

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