Chemically derived hepatic progenitors (CdHs)Ex vivo gene therapyLiver progenitorsRegenerative medicineTransplantationEx vivo gene therapy through convergence study with progenitors and base/prime editors provides valuable approaches that can be utilized in the study and treatment of hereditary intractable ...
therapeutic transgenes can be inserted in a safe and highly transcribed locus to maximize protein expression. Here, we describe an ex vivo editing approach to achieve efficient gene targeting in human hematopoietic
In vivo gene editing after oviduct injection with rAAV vectors.aSchematic representation of the strategy to induce in vivo gene editing of theTyrlocus. rAAV vectors carryingSpCas9and sgTyrexpression constructs were injected directly into the oviduct of plugged C57BL/6NJ females mated to C57BL/6N...
Ex vivo gene editing and cell therapy for hereditary tyrosinemia type 1 hydroxyphenylpyruvate dioxygenase (HPD) to rescue mice deficient in fumarylacetoacetate hydrolase (FAH), a disorder known as hereditary tyrosinemia type 1 (... I Ates,C Stuart,Rathbone, TannerBarzi, MercedesHe, GordonMajor, ...
A therapeutic option for monogenic disorders is gene therapy with ex vivo-transduced autologous hematopoietic stem cells (HSCs). Safety or efficacy studies of ex vivo-modified HSCs are conducted in humanized mouse models after ablation of the murine bone marrow and transfer of human CD34+ HSCs. En...
Until recently, progress in ex vivo gene therapy (GT) for human immunodeficiency virus-1 (HIV-1) treatment has been incremental. Long-term HIV-1 remission in a patient who received a heterologous stem cell transplant for acquired immunodeficiency syndrome-related lymphoma from a CCR5(-/-) donor...
Sickle-cell disease (SCD) is caused by an A·T-to-T·A transversion mutation in the β-globin gene (HBB). Here we show that prime editing can correct the SCD allele (HBBS) to wild type (HBBA) at frequencies of 15%–41% in haematopoietic stem and progenit
Ex vivo gene therapy, a technique where genetic manipulation of cells is undertaken remotely and more safely since it is outside the body, is an emerging therapeutic strategy particularly well suited to targeting a specific organ rather than for treating a whole organism. The eye and visual pathw...
Ex vivo gene therapy using targeted engraftment of NGF-expressing human NT2N neurons attenuates cognitive deficits following traumatic brain injury in mice. 来自 NCBI 喜欢 0 阅读量: 71 作者: L Longhi,DJ Watson,KE Saatman,HJ Thompson,C Zhang,S Fujimoto,N Royo,D Castelbuono,R Raghupathi,JQ ...
We first cultured CB CD34+ cells on telomerase gene-... A Fujimi,T Matsunaga,M Kobune,... - 《International Journal of Hematology》 被引量: 142发表: 2008年 Ex-vivo generation of human red cells for transfusion The present article reviews the recent data concerning the generation of red ...