Molecular Therapy
Papers 9929
1 page of 993 pages (9,929 results)
#1Veronica Gough (Duke University)
#2Charles A. Gersbach (Duke University)H-Index: 39
#1Shiwani Agarwal (Paul Ehrlich Institute)H-Index: 1
#2Julia D. S. Hanauer (Paul Ehrlich Institute)H-Index: 1
Last. Christian J. Buchholz (Goethe University Frankfurt)H-Index: 1
view all 6 authors...
Abstract T cells modified with CD19-specific chimeric antigen receptors (CARs) result in significant clinical benefit for leukemia patients but constitute a challenge for manufacturing. We have recently demonstrated the in vivo generation of CD19-CAR T cells using the CD8-targeted lentiviral vector CD8-LV. Here we investigated the in vivo generation of CD4-positive CAR T cells using CD4-targeted LV (CD4-LV). Administration of CD4-LV into NSG mice transplanted with human PBMC led to 40 - 60% of h...
#1Kentaro Yamada (IUPUI: Indiana University – Purdue University Indianapolis)
#2Roland W. Herzog (IUPUI: Indiana University – Purdue University Indianapolis)H-Index: 2
#1Aurore Besse (University of Paris)H-Index: 2
#2Stéphanie Astord (University of Paris)H-Index: 2
Last. Maria Grazia Biferi (University of Paris)H-Index: 2
view all 10 authors...
Abstract Spinal muscular atrophy (SMA) is a neuromuscular disease mainly caused by mutations or deletions in the survival of motor neuron (SMN1) gene and characterized by the degeneration of motor neurons and progressive muscle weakness. A viable therapeutic approach for SMA patients is a gene replacement strategy that restores functional SMN expression using adeno-associated virus serotype 9 vectors (AAV9). Currently, systemic or intra-cerebrospinal fluid (intra-CSF) delivery of AAV9-SMN are be...
#1Zhan LiH-Index: 1
Last. Xiang AoH-Index: 1
view all 14 authors...
Abstract Sepsis is potentially a life-threatening condition and a major cause of death in the intensive care units (ICUs), which is characterized by multiple organ dysfunctions as a result of unbalanced host inflammatory response to pathogens. However, effective treatment or intervention to prevent sepsis-associated lethality is still lacking. Human umbilical cord mesenchymal stem cells (hUC-MSCs) transplantation has been shown to have potent immunomodulatory properties and improve tissue repair...
#1Colby R. Maldini (UPenn: University of Pennsylvania)H-Index: 2
#2Kevin Gayout (UPenn: University of Pennsylvania)
Last. James L. Riley (UPenn: University of Pennsylvania)H-Index: 63
view all 8 authors...
Abstract HIV infection preferentially depletes HIV-specific CD4+ T cells thereby impairing antiviral immunity. Here, we explore the therapeutic utility of adoptively transferred CD4+ T cells expressing an HIV-specific Chimeric Antigen Receptor (CAR4) to restore CD4+ T cell function to the global HIV-specific immune response. We demonstrated that CAR4 T cells directly suppressed in vitro HIV replication and eliminated virus-infected cells. Notably, CAR4 T cells containing intracellular domains (I...
#1Ion CristóbalH-Index: 14
#2Melani LuqueH-Index: 2
Last. Jesús García-FoncillasH-Index: 28
view all 5 authors...
#1Kathleen A. Christie (Ulster University)H-Index: 3
#2Louise J. Robertson (Ulster University)
Last. M. Andrew Nesbit (Ulster University)H-Index: 23
view all 11 authors...
Abstract CRISPR-Cas9 provides a tool to treat autosomal dominant disease by NHEJ gene disruption of the mutant allele. In order to discriminate between wild-type and mutant alleles, SpCas9 must be able to detect a single nucleotide change. Allele-specific editing can be achieved by employing either a guide-specific approach, in which the missense mutation is found within the guide sequence; or a PAM-specific approach, in which the missense mutation generates a novel PAM. While both approaches ha...
#1Lei Yang (ECNU: East China Normal University)H-Index: 2
#2Liren Wang (ECNU: East China Normal University)H-Index: 8
Last. Honghui HanH-Index: 9
view all 17 authors...
Abstract Base editing technology efficiently generates nucleotide conversions without inducing excessive double-strand breaks (DSBs), which makes it a promising approach for genetic disease therapy. In this study, we generated a novel hereditary tyrosinemia type 1 (HT1) mouse model, which contains a start codon mutation in the fumarylacetoacetate hydrolase (Fah) gene by using an adenine base editor (ABE7.10). To investigate the feasibility of base editing for recombinant adeno-associated virus (...
Top fields of study
Genetic enhancement
Molecular biology
Viral vector