Results 241 to 250 of about 1,210,893 (313)

Versatile Cell Penetrating Peptide for Multimodal CRISPR Gene Editing in Primary Stem Cells

Advanced Functional Materials, EarlyView.
CRISPR machinery in diverse molecular formats (DNA, RNA, and ribonucleic protein) is complexed into nanoparticles with the cell‐friendly arginine‐alanine‐leucine‐alanine (RALA) cell‐penetrating peptide. Nanoparticles are delivered to primary mesenchymal stem cells ex vivo or locally in vivo to facilitate multimodal CRISPR gene editing. This RALA‐CRISPR
Joshua P. Graham, Jose Gabriel Castro, Lisette C. Werba, Joseph Amitrano, Luke Fardone, Kevin P. Francis, Anand Ramamurthi, Michael J. Layden, Helen O. McCarthy, Tomas Gonzalez‐Fernandez   +9 more
wiley   +1 more source

O26: Analysis of patient-reported outcomes and a functional assessment from 3-year nitisinone treatment trial in patients with alkaptonuria

Genetics in Medicine Open
Kathryn Spears, Francis Rossignol, Monique Perry, Joy Bryant, William Gahl, Wendy Introne   +5 more
doaj   +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

Preventive Human Genome Editing and Enhancement: Candidate Criteria for Governance. [PDF]

Hastings Cent Rep
Juengst E, Flatt MA, Conley JM, Davis A, Henderson G, MacKay D, Major R, Walker RL, Cadigan RJ.   +8 more
europepmc   +1 more source

Endocytic Programming via Porous Silicon Nanoparticles Enhances TLR4 Nanoagonist Potency for Macrophage‐Mediated Immunotherapy

Advanced Functional Materials, EarlyView.
Porous silicon nanoparticles (PSiNPs) reprogram macrophage endocytosis of manganese@albumin‐based TLR4 nanoagonists, driving TRIF‐biased TLR4 signaling, eliciting robust proinflammatory responses, and potentiating macrophage‐mediated immunotherapeutic effects against NSCLC.
Xiaomei Zhang, Huiying Li, Shuodan Huang, Linxuan Zhang, Yan Gao, Ruoran Wang, Xiaoyu Wang, Hélder A. Santos, Zhenyu Yin, Bing Xia   +9 more
wiley   +1 more source

Replicative DNA polymerase epsilon and delta holoenzymes show wide-ranging inhibition at G-quadruplexes in the human genome. [PDF]

Nucleic Acids Res
Hile SE, Weissensteiner MH, Pytko KG, Dahl J, Kejnovsky E, Kejnovská I, Hedglin M, Georgakopoulos-Soares I, Makova KD, Eckert KA.   +9 more
europepmc   +1 more source

FeDSNP‐Pa Nanoassemblies: A Triple‐Action Therapeutic Strategy Targeting Oxidative Stress, Inflammation, and Pyroptosis for Retinal Ganglion Cell Protection in Glaucoma

Advanced Functional Materials, EarlyView.
FeDSNP‐Pa, a metallized nanoparticle loaded with sodium pyruvate (Pa), exerts triple therapeutic effects by scavenging reactive oxygen species (ROS), suppressing inflammatory responses, and inhibiting pyroptosis signaling pathways. This multifunctional neuroprotective strategy protecting retinal ganglion cells (RGCs) from elevated intraocular pressure ...
Yukun Wu, Xue Li, Tong Wu, Dan Ji, Yugang Bai, Xuezhi Zhou   +5 more
wiley   +1 more source

Functional characteristics and computational model of abundant hyperactive loci in the human genome. [PDF]

Elife
Hudaiberdiev S, Ovcharenko I.
europepmc   +1 more source

A Study of Nucleosome Sequence and Relative Position to Gene Transcription Start Sites in Yeast and Human Genomes

, 2014
Greg Wright
openalex   +1 more source

Next‐Generation Bio‐Reducible Lipids Enable Enhanced Vaccine Efficacy in Malaria and Primate Models

Advanced Functional Materials, EarlyView.
Structure–activity relationship (SAR) optimization of bio‐reducible ionizable lipids enables the development of highly effective lipid nanoparticle (LNP) mRNA vaccines. Lead LNPs show superior tolerability and antibody responses in rodents and primates, outperforming approved COVID‐19 vaccine lipids.
Ruben De Coen, Sabah Kasmi, Shrinivas Dumbre, Lies Baekens, Elise Seynaeve, Louise Van den Hauwe, Niccolò Cantini, Aello Garantoudi, Filipe Branco Madeira, Alain Marrot, Emma Tuypens, Sofie Seghers, Anna Kuchmiy, Elisabeth Brabants, Bart Vanderborght, Ine Maes, Sarah Vandenberghe, Jessica Filtjens, Alícia Gordún Peiró, Daliya Kancheva, Ayla Debraekeleer, Florence Lambolez, Emily De Lombaerde, Yong Chen, Rafael Miyazawa Martins, Pauline Formaglio, Damya Laoui, Sofie Deschoemaeker, Bruno G. De Geest, Rogerio Amino, Stefaan De Koker   +30 more
wiley   +1 more source