Results 211 to 220 of about 1,037,544 (299)

Therapeutic base editing to generate a gain-of-function F9 variant for hemophilia B.

Blood
Baatartsogt N, Kashiwakura Y, Hiramoto T, Ito R, Sato R, Nagao Y, Naruoka H, Takata H, Hayakawa M, Batjargal K, Togashi T, Hoshino A, Shimizu T, Sato Y, Ishida T, Nureki O, Ohmori T.   +16 more
europepmc   +1 more source

Mini-Catalytically Inactive Cas13X-Derived RNA Base Editing of β-Catenin Attenuates Pulmonary Damage in a Murine Acute Lung Injury Model. [PDF]

MedComm (2020)
Liu W, Bi W, Hou S, Du J, Zeng L, Zhang A, Zhang H, Wen D, Cai Q, Gao C, Lin P, Wu M, Li L, Jiang J.   +13 more
europepmc   +1 more source

Proteasomal degradation of intracellularly expressed Amblyomin‐X limits suicide gene therapy potential in melanoma cells

FEBS Open Bio, EarlyView.
This study explores the feasibility of expressing the antitumoral protein Amblyomin‐X through a suicide gene therapy approach and investigates its intracellular fate after gene delivery. Although the gene is efficiently expressed, melanoma cells rapidly degrade the Amblyomin‐X protein via proteasome activity.
Victor Dal Posolo Cinel, Carlos DeOcesano Pereira, Aline Ramos Maia Lobba, Melissa Regina Fessel, Ana Marisa Chudzinski‐Tavassi   +4 more
wiley   +1 more source

Coiled-coil heterodimer-mediated split base editing systems enable flexible and robust nucleotide substitutions. [PDF]

Nat Commun
Mu S, Li Q, Chen M, Li Z, Ma Y, Li Y, Song Y, Hou S, Ding Y, Ju J, Lin Y, Zhang J, Yang Y, Ren X, Li N, Jin Q, Lai L, Wang K, Shi H.   +18 more
europepmc   +1 more source

MiR‐513a promotes human erythroid differentiation by modulating c‐Jun

FEBS Open Bio, EarlyView.
During early human erythropoiesis, miR‐513a promoted erythroid differentiation in primary human CD34+ hematopoietic stem‐progenitor cells and human TF‐1 erythroleukemic cells by indirectly decreasing c‐Jun and phospho‐c‐Jun expression, which are associated with increased GATA1 expression.
MinJung Kim, Brittany Taylor, Shannon Bolten, Christian L. Eberly, Mahliya Abdurahman, T. Michael Creed, Acong Yang, Taylor L. Hatchet, Tristan Dyson, Shuo Gu, Curt I. Civin, Tami J. Kingsbury   +11 more
wiley   +1 more source

LNP-mediated in vivo base editing corrects Agxt to cure primary hyperoxaluria type 1. [PDF]

Clin Transl Med
Zhang D, Zheng R, Chen Z, Chen X, Yang L, Huo Y, Zhao Y, Huang J, Zhang D, Yin S, Li D, Geng H.   +11 more
europepmc   +1 more source

Decreased cold‐sensing function of the transient receptor potential channel TRPM8 from tailed amphibians

FEBS Open Bio, EarlyView.
Despite frogs avoiding low temperatures, examination of four salamander species revealed that none avoided cold and all possessed cold tolerance. Functional analysis of TRPM8, a cold sensor, showed that all salamander TRPM8s had lost their cold sensitivity.
Tadahiro Sawao, Daiki Kobayashi, Shogo Hori, Osamu Saitoh   +3 more
wiley   +1 more source

Noncanonical target-strand cytosine base editing via engineered Un1Cas12f1 platform. [PDF]

Nat Commun
Song Z, Guo J, Fan Z, Huang S, Li G, Zhao Z, Chen B, Huang S, Zheng W, Wei Y, Chen Y, Huang X, Liu J, Wu L, Wang X.   +14 more
europepmc   +1 more source

Digital twins to accelerate target identification and drug development for immune‐mediated disorders

FEBS Open Bio, EarlyView.
Digital twins integrate patient‐derived molecular and clinical data into personalised computational models that simulate disease mechanisms. They enable rapid identification and validation of therapeutic targets, prediction of drug responses, and prioritisation of candidate interventions.
Anna Niarakis, Philippe Moingeon
wiley   +1 more source