Results 121 to 130 of about 11,317 (250)
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
Journal of Inherited Metabolic Disease, Volume 49, Issue 2, March 2026.ABSTRACT Fabry disease is an X‐linked lysosomal storage disorder caused by mutations in the GLA gene, leading to deficient α‐galactosidase A (α‐Gal A) activity and pathological accumulation of globotriaosylceramide (Gb3) and globotriaosylsphingosine (Lyso‐Gb3) in various organs.
Siwu He +15 more
wiley +1 more source
, 2017 This article answers the question: My patient carries a diagnosis of chronic anemia and has been treated for irondeficiency in the past with minimal to no improvement.
Coberly, Emily, Ringling, Rebecca
core
American Journal of Hematology, Volume 101, Issue 3, Page 628-632, March 2026.
Zhenbin Wei +19 more
wiley +1 more source
Clinical Characteristics and Gene Mutations of Hereditary Spherocytosis in 59 Chinese Children
Molecular Genetics &Genomic Medicine, Volume 14, Issue 3, March 2026.Summary of the causative genes' spectrum of 59 Chinese HS Children patients. The schematic diagram of ankyrin, β‐spectrin, α‐spectrin, and band three protein domains with ANK1, SPTB, SPTA1, and SLC4A1 mutations. Distribution of mutations in the exons of ANK1 and SPTB genes.
Yuzhuopu Li +7 more
wiley +1 more source
Simultaneous occurrence of advanced neuroblastoma and acute lymphoblastic leukemia: a case report [PDF]
, 2011 Neuroblastoma is the most common extracranial solid tumor in children. The cooccurrence of neuroblastoma and acute lymphoblastic leukemia is rare. We report a rare case of advanced-stage neuroblastoma in a 3.5-year-old girl that was accompanied by ...
Fekri, Kiavash. +4 more
core +1 more source
CPT: Pharmacometrics &Systems Pharmacology, Volume 15, Issue 3, March 2026.ABSTRACT Despite the fact that modeling and simulation are now recognized as promising innovative methodologies, their use in the context of development of drugs for sickle cell disease and Thalassemia has not yet been reviewed. Considering the challenges of conducting clinical trials for hemoglobinopathies, our work aims at exploring the current ...
Grace Shalom Govere +2 more
wiley +1 more source
Bases moleculares de hemoglobinopatias na Argentina [PDF]
, 2017 Durante el desarrollo de un individuo se expresan distintas cadenas de globina de tipo α y no-α, que se combinan en tetrámeros para formar hemoglobina. Los genes que las codifican se organizan en familias.
Scheps, Karen, Varela, Viviana
core
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
Identification of the High-Risk Gravida [PDF]
, 1980 In the past, there has been considerable pessimism about our ability to identify the pregnant patient at risk. However, with the development of sophisticated diagnostic techniques these patients can be identified and with appropriate treatment their ...
Coogan, Emily M., Petres, Robert E.
core +1 more source