Results 231 to 240 of about 2,044,942 (306)

P192: CRISPR-mediated base editing of the Irish infantile Tay-Sachs mutation

Genetics in Medicine Open
Madison Hernandez, Cynthia Tifft
doaj   +1 more source

Prediction of target genes and functional types of cis-regulatory modules in the human genome reveals their distinct properties. [PDF]

BMC Biol
Yuan S, Ni P, Su Z.
europepmc   +1 more source

Lipid Nanoparticles for the Delivery of CRISPR/Cas9 Machinery to Enable Site‐Specific Integration of CFTR and Mutation‐Agnostic Disease Rescue

Advanced Functional Materials, EarlyView.
Lipid nanoparticles (LNPs) are optimized to co‐deliver Cas9‐encoding messenger RNA (mRNA), a single guide RNA (sgRNA) targeting the endogenous cystic fibrosis transmembrane conductance regulator (CFTR) gene, and homologous linear double‐stranded donor DNA (ldsDNA) templates encoding CFTR.
Ruth A. Foley, Paul G. Ayoub, Vrishti Sinha, Colin Juett, Alicia Sanoyca, Emily C. Duggan, Lindsay E. Lathrop, Priyanka Bhatt, Kevin Coote, Beate Illek, Brigitte N. Gomperts, Donald B. Kohn, Steven J. Jonas   +12 more
wiley   +1 more source

Polymorphic pseudogenes in the human genome - a comprehensive assessment. [PDF]

Hum Genet
Lopes-Marques M, Peixoto MJ, Cooper DN, Prata MJ, Azevedo L, Castro LFC.   +5 more
europepmc   +1 more source

Combinatorial Synthesis of Next Generation Water‐Soluble Quaternized N‐Halamine Oligomers with Long‐Lasting Antiviral Properties

Advanced Functional Materials, EarlyView.
A combinatorial library of dual‐functional antiviral oligomers incorporating N‐halamine and quaternary ammonium functionalities is developed for long‐lasting antiviral activity. The lead materials exhibit rapid and durable antiviral activity against SARS‐CoV‐2 variants and influenza H1N1, with 4 to 5 log reduction in viral copies at 5 mg mL−1 ...
Eid Nassar‐Marjiya, Konda Reddy Kunduru, Krishanu Ghosal, Luna Rizik, Nofar Atari, Mahran Daifa, Neta Kutner, Bassma Khamaisi, Qi Wu, Simran Jindal, Merna Shaheen‐Mualim, Ranabir Majumder, Mahitosh Mandal, Michal Mandelboim, Shady Farah   +14 more
wiley   +1 more source

Integration of therapeutic cargo into the human genome with programmable type V-K CAST. [PDF]

Nat Commun
Liu J, Aliaga Goltsman DS, Alexander LM, Khayi KK, Hong JH, Dunham DT, Romano CA, Temoche-Diaz MM, Chadha S, Fregoso Ocampo R, Oki-O'Connell J, Janson OP, Turcios K, Gonzalez-Osorio L, Muysson J, Rahman J, Laperriere SM, Devoto AE, Castelle CJ, Butterfield CN, Cost GJ, Brown CT, Thomas BC.   +22 more
europepmc   +1 more source

Gender, genes and genetics: from Darwin to the human genome [PDF]

, 2000
Segal, Lynne
core  

RNA‐Micelles as Self‐Assembling Structures for Efficient Co‐Delivery of Synergistic siRNA and Nucleoside Analogues to Treat CRC Lung Metastasis

Advanced Functional Materials, EarlyView.
Two kinds of self‐assembled RNA micelles were used to co‐deliver synergistic siRNA and nucleoside analogues for the treatment of colorectal cancer lung metastases. Near‐complete elimination of lung cancer metastases was confirmed in an orthotopic lung metastasis model constructed using human colorectal cancer lung metastases patient surgical samples to
Kai Jin, Piotr Rychahou, Daniel W. Binzel, B. Mark Evers, Peixuan Guo   +4 more
wiley   +1 more source

Ribosomal DNA arrays are the most H-DNA rich element in the human genome. [PDF]

NAR Genom Bioinform
Chantzi N, Chan CSY, Patsakis M, Nayak A, Montgomery A, Mouratidis I, Georgakopoulos-Soares I.   +6 more
europepmc   +1 more source

Controlling 3D Contractility via Engineered Fibrous Hydrogel Composites

Advanced Functional Materials, EarlyView.
A tunable composite contractile assembly (CCA) is developed that can permit or resist contraction without changing cell‐adhesion and density, unlike collagen whose inherent coupling of these ECM cues limits its use as a platform in contractility studies.
Karen L. Xu, Yuqi Zhang, Alysse DeFoe, Georgios Kotsaris, Brendan Stoeckl, Matthew D. Davidson, Jason A. Burdick, Robert L. Mauck   +7 more
wiley   +1 more source