Results 211 to 220 of about 83,579 (259)

Epilepsy‐associated SCN2A‐L1342P mutation drives network hyperexcitability and widespread transcriptomic changes in human cortical organoids

open access: yesEpilepsia, Volume 67, Issue 10, Page 5514-5530, October 2026.
Abstract Objective SCN2A pathogenic mutations, such as the recurrent heterozygous Nav1.2‐L1342P, are monogenic causes of epilepsy. In this human‐induced pluripotent stem cell–derived model system, we aim to investigate the molecular and cellular mechanisms underlying SCN2A‐L1342P‐associated pathology. Methods Using a human male induced pluripotent stem
Maria I. Olivero‐Acosta   +26 more
wiley   +1 more source

Human erythroid models of sickle cell disease

open access: yesHemaSphere, Volume 10, Issue 10, October 2026.
Abstract Sickle cell disease (SCD) is a group of severe inherited red blood cell disorders causing a major global healthcare burden. Despite recent advances in gene therapy and stem cell transplantation, treatment options remain very limited, highlighting the urgent need for the development of new therapeutic approaches.
Deborah E. Daniels   +5 more
wiley   +1 more source

Heparin Can Augment Extracellular Vesicle Yield by Inhibiting Reuptake in Donor Cells During Cell Propagation

open access: yesJournal of Extracellular Vesicles, Volume 15, Issue 10, October 2026.
Heparin augments EV yield in culture by inhibiting EV binding to cell‐surface HSPG and masking EV surface. Preventing autocrine and homotypic paracrine EV uptake during cell culture results in EV accumulation and higher yield. Heparin improved EV recovery after storage in NaCl but not in complex media or protein‐rich buffer(Please see Figure 9 for ...
André Cronemberger Andrade   +13 more
wiley   +1 more source

CRISPR/Cas9‐Mediated Correction of the SLC25A38 Mutation in a Patient With Sideroblastic Anemia

open access: yesThe Journal of Gene Medicine, Volume 28, Issue 10, October 2026.
In this study, the identity of fibroblast cells isolated from the patient was identified using several methods. Then, the sgRNA3‐pX459 plasmid together with ssODN was transfected into fibroblast cells with a transfection efficiency of 49.64%. Finally, the entry of ssODN donor into fibroblast cells was confirmed using PCR‐RFLP and PAGE methods. ABSTRACT
Shokoufeh Yazdanparast   +4 more
wiley   +1 more source

Extracellular Vesicles: Classification, Biological Functions, Diseases, and Therapeutic Opportunities

open access: yesMedComm, Volume 7, Issue 10, October 2026.
• EV content maintains cellular homeostasis and is involved in several disease onset and progression. • miRNA export in EVs depends on: RNA‐binding proteins recognizing motif sequences and on epitranscriptomic modifications. • The disclosure of delivery mechanisms is crucial for developing personalized EV‐based therapeutic tools. ABSTRACT Extracellular
Sabrina Garbo   +4 more
wiley   +1 more source

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