Results 131 to 140 of about 35,703 (223)
Therapeutic Gene Editing: DNA Repair Pathways, Emerging Editors, and Clinical Progress
iNew Medicine, Volume 2, Issue 1, March 2026.ABSTRACT The field of gene editing has evolved rapidly over the past decade, progressing from programmable zinc‐finger nucleases (ZFNs) and transcription activator‐like effector nucleases (TALENs) to the widespread adoption of CRISPR‐Cas systems. First‐generation editors catalyzed genome engineering by introducing targeted double‐strand breaks (DSBs ...
Li‐Kuang Tsai +7 more
wiley +1 more source
RNA‐Based Therapies for Inherited Metabolic Disorders
Journal of Inherited Metabolic Disease, Volume 49, Issue 2, March 2026.ABSTRACT Inherited metabolic disorders (IMDs) are a diverse and complex group of genetic conditions resulting from deficiencies in enzymes, transporters, or cofactors. These deficiencies lead to metabolic dysfunction and severe clinical consequences. Despite significant progress in understanding their molecular basis, treatment options remain limited ...
Reddy Sreekanth Vootukuri +5 more
wiley +1 more source
Journal of Dermatological TreatmentBackground Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) not only regulates cholesterol metabolism and cardiovascular disorder but also modifies inflammatory response and autoimmunity.Objective This study investigated the relation of PCSK9 to ...
Xuwen Yin +5 more
doaj +1 more source
The Grey Zone Project: Risk‐Based Classification of ABCD1 Variants in X‐Linked Adrenoleukodystrophy
Journal of Inherited Metabolic Disease, Volume 49, Issue 2, March 2026.ABSTRACT Newborn screening (NBS) for X‐linked adrenoleukodystrophy (ALD) enables early identification of boys at risk for adrenal insufficiency (AI) and cerebral ALD (CALD). However, NBS frequently identifies ABCD1 variants of uncertain significance (VUS), which are associated with only borderline‐elevated C26:0‐lysophosphatidylcholine (LPC(26:0 ...
Troy C. Lund +17 more
wiley +1 more source
Nature Inspired Delivery Vehicles for CRISPR‐Based Genome Editing
Small, Volume 22, Issue 16, 17 March 2026.The review highlights nature‐inspired nanocarriers for CRISPR delivery, emphasizing viral vectors, extracellular vesicles, liposomes, and lipid nanoparticles. It discusses their roles in improving specificity, minimizing immunogenicity, and overcoming barriers in genome editing. Recent advancements, challenges, and therapeutic applications are explored,
Elizabeth Maria Clarissa +4 more
wiley +1 more source
Insights Into Macrophage Polarization and M1/M2 Balance in Diabetic Foot Ulcers
Journal of Diabetes, Volume 18, Issue 3, March 2026.This graphical abstract illustrates pharmaceutical agents that modulate M1/M2 macrophage polarization in diabetic foot ulcers (DFUs). M1 macrophages (red) drive inflammation, whereas M2 macrophages (blue) promote tissue repair. In DFUs, persistent M1 predominance contributes to chronic non‐healing. Five key agents targeting this imbalance are depicted:
Jing Zhang +6 more
wiley +1 more source
PLoS ONE, 2022 Prescription of PCSK9-inhibitors has increased in recent years but not much is known about its off-target effects. PCSK9-expression is evident in non-hepatic tissues, notably the brain, and genetic variation in the PCSK9 locus has recently been shown to ...
Alisha Aman +7 more
doaj
FOURIER & PCSK9 RNAi: Towards enhancing durability and efficacy of PCSK9 inhibitors
Global Cardiology Science and Practice, 2017 [first paragraph of article]The 2003 discovery of proprotein convertase subtilisin–kexin type-9 (PCSK9) – a circulating protein targeting the low density lipoprotein (LDL) receptor for its degradation – paved the way for the development of several therapeutic approaches which inhibit the protein itself (by monoclonal antibodies) or its RNA (by RNA ...
openaire +3 more sources
The discovery of PCSK9 inhibitors: A tale of creativity and multifaceted translational research. [PDF]
, 2013 Elguindy, A, Yacoub, MH
core +1 more source