Turnitin
降AI改写
早检测系统
早降重系统
Turnitin-UK版
万方检测-期刊版
维普编辑部版
Grammarly检测
Paperpass检测
checkpass检测
PaperYY检测
Gene Therapy for Sickle Cell Disease: Recent Advances, Clinical Trials, and Future directions
J Ballantine, JF Tisdale - Cytotherapy, 2024 - Elsevier
Abstract Sickle Cell Disease (SCD) is the most common inherited blood disorder worldwide,
impacting millions and imposing severe healthcare challenges, particularly in resource …
impacting millions and imposing severe healthcare challenges, particularly in resource …
Preclinical Efficacy of a Modified Gamma Globin Lentiviral Vector Mediated Gene Therapy in Berkeley Sickle Cell Anemia Mouse and Human Xenograft Models
A Shrestha, DM Pillis, S Felker, M Chi, K Wagner… - … Therapy Methods & …, 2025 - cell.com
We previously showed correction of sickle cell anemia (SCA) in mice utilizing a lentiviral
vector (LV) expressing human γ-globin. Herein, we made a G16D mutation in the γ-globin …
vector (LV) expressing human γ-globin. Herein, we made a G16D mutation in the γ-globin …
[HTML][HTML] The Optimized γ-Globin Lentiviral Vector GGHI-mB-3D Leads to Nearly Therapeutic HbF Levels In Vitro in CD34+ Cells from Sickle Cell Disease Patients
We have previously demonstrated that both the original γ-globin lentiviral vector (LV) GGHI
and the optimized GGHI-mB-3D LV, carrying the novel regulatory elements of the 3D HPFH …
and the optimized GGHI-mB-3D LV, carrying the novel regulatory elements of the 3D HPFH …
Large-scale discovery of potent, compact and lineage specific enhancers for gene therapy vectors
Regulation of gene expression during cell development and differentiation is chiefly
orchestrated by distal noncoding regulatory elements that precisely modulate cell selective …
orchestrated by distal noncoding regulatory elements that precisely modulate cell selective …
[PDF][PDF] Identification of genome safe harbor loci for human gene therapy based on evolutionary biology and comparative genomics
MA Quezada-Ramírez, S Loncar, MA Campbell… - bioRxiv, 2023 - researchgate.net
Gene transfer into hematopoietic stem and progenitor cells (HSPCs) involving
indiscriminately integrating viral vectors has unpredictable outcomes including potential …
indiscriminately integrating viral vectors has unpredictable outcomes including potential …
Development of evolutionarily conserved viral integration sites as safe harbors for human gene therapy
MA Quezada-Ramírez, S Loncar, MA Campbell… - bioRxiv, 2023 - biorxiv.org
Gene transfer into CD34+ hematopoietic stem and progenitor cells (HSPCs) involving
integrating viral vectors has unpredictable outcomes including potential adverse events like …
integrating viral vectors has unpredictable outcomes including potential adverse events like …