Results 131 to 140 of about 362,296 (275)

Lipid Nanoparticles for Delivery of CRISPR Gene Editing Components

open access: yesSmall Methods, EarlyView.
The review presents a comprehensive overview of each component of lipid nanoparticles(LNPs)and their effects on editing efficiency. It specifically highlights strategies for achieving non‐liver delivery, aiming for broader applications in gene editing. Furthermore, this review summarizes the applications of LNPs in gene editing and offers insights for ...
Fan Wu   +6 more
wiley   +1 more source

Duchenne muscular dystrophy

open access: yesNature Reviews Disease Primers, 2017
Sophia Roberts
semanticscholar   +1 more source

Spasmodic Dysphonia

open access: yesWorld Journal of Otorhinolaryngology - Head and Neck Surgery, EarlyView.
ABSTRACT Spasmodic dysphonia is a laryngeal dystonia that can present as adductor, abductor, or mixed types, with or without tremor. The etiology is not understood fully. Comprehensive evaluation is required to establish the diagnosis. Treatments include voice therapy, medications, botulinum toxin injection, laryngeal surgery, deep brain stimulation ...
Aaron J. Jaworek, Robert T. Sataloff
wiley   +1 more source

Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy

open access: yesScience, 2016
Chengzu Long   +9 more
semanticscholar   +1 more source

The Swedish National Pediatric Cataract Register (PECARE): Coexisting systemic disorders 2007–2023

open access: yesActa Ophthalmologica, EarlyView.
Abstract Purpose To analyse the frequency and type of coexisting systemic disorders in children operated on for cataract in Sweden. Methods Data were retrieved from the Swedish National Pediatric Cataract Register (PECARE) for children operated between January 1, 2007, and December 31, 2023 (n = 975), including follow‐ups at age 1, 2, 5 and 10 ...
David Wackerberg   +9 more
wiley   +1 more source

Laypeople's Views on the Narrative Identity and Societal Treatment of Genetically Modified People

open access: yesBioethics, EarlyView.
ABSTRACT Genome editing in human embryos could raise new ethical issues by changing future people's narrative and numerical identity. Most philosophers agree that some genetic modifications would have larger effects on identity than others, but they disagree on what criteria might explain these differences and have not supported their claims ...
Derek So, Yann Joly, Robert Sladek
wiley   +1 more source

Confronting medical grifting: Fraudulent and unproven products and interventions in apheresis, transfusion and biotherapies

open access: yesBritish Journal of Haematology, EarlyView.
A basic overview of medical grifting for haematologists with practical guidance in identification and tips for management. Summary Medical scams or grifting has long been a societal issue, though in recent years, the problem has become increasingly mainstream, especially as it relates to transfusion medicine, apheresis and biotherapies.
Brian D. Adkins   +7 more
wiley   +1 more source

Biallelic COL4A2 Variants Associated With Brain Small Vessel Disease and Brain Malformations

open access: yesClinical Genetics, EarlyView.
Biallelic COL4A2 variants cause a spectrum of brain abnormalities such as brain small vessel disease (BSVD). We describe two cases—one with cerebrovascular disruption and one with cortical malformations—expanding the recessive phenotype. Protein modeling reveals destabilization by p.(Arg179Cys), reinforcing its pathogenic role and highlighting collagen
Anees Muhammad   +11 more
wiley   +1 more source

PTRF Confers Melanoma‐Acquired Drug Resistance Through the Upregulation of EGFR

open access: yesCell Proliferation, EarlyView.
The upregulation of PTRF, a critical caveolar component, and the concomitant increase in caveolae number potentiate cellular proliferation and survival pathways, thereby contributing to melanoma drug resistance. ABSTRACT Melanoma is the most serious type of skin cancer. About half of all melanomas have activating BRAF mutations.
Miao Wang   +11 more
wiley   +1 more source

LCN2‐ACOD1 Signalling Affects the Post‐Injury Regeneration of Skeletal Muscle Through Mediating Ferroptosis

open access: yesCell Proliferation, EarlyView.
LCN2 mediates the suppression of C2C12 myoblast proliferation and myotube formation via erastin‐induced ferroptosis. Mechanistically, the LCN2‐ACOD1 axis regulates skeletal muscle growth via mitochondria‐associated ferroptosis, providing a theoretical basis for the prevention and therapy of muscle‐related diseases.
Xiaojing Hao   +12 more
wiley   +1 more source

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