Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

Correction of murine Bernard-Soulier syndrome by lentivirus-mediated gene therapy.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Mar 2012

Kanaji Sachiko, Kuether Erin L, Fahs Scot A, Schroeder Jocelyn A, Ware Jerry, Montgomery Robert R, Shi Qizhen

Abstract excerpt

Bernard-Soulier syndrome (BSS) is an inherited bleeding disorder caused by a defect in the platelet glycoprotein (GP) Ib-IX-V complex. The main treatment for BSS is platelet transfusion but it is often limited to severe bleeding episodes or surgical interventions due to the risk of alloimmunization. We have previously reported successful expression of human GPIbα (hGPIbα) in human megakaryocytes using a...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to Antibodies
Share to Bernard-Soulier Syndrome

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Blood Platelets

Share to Bone Marrow Transplantation

Share to Hemorrhage

Share to Lentivirus

Share to Platelet Glycoprotein GPIb-IX Complex

Share to Thrombocytopenia

Source

Open in PubMed Open full text

Identifiers

PMID: 22044935
DOI: 10.1038/mt.2011.231
PMCID: PMC3293608

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 5 Sept 2026. Manifest publication-graph-cohort-2026-09-05.1.

Source receipt: pubmed, retrieved 5 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Correction of murine Bernard-Soulier syndrome by lentivirus-mediated gene therapy.PMID 22044935Read ↗

Non-myeloablative conditioning with busulfan before hematopoietic stem cell transplantation leads to phenotypic correction of murine Bernard-Soulier syndrome.Semantic proximity Read ↗

Lentiviral gene therapy reverts GPIX expression and phenotype in Bernard-Soulier syndrome type CSemantic proximity Read ↗

In vivo enrichment of genetically manipulated platelets corrects the murine hemophilic phenotype and induces immune tolerance even using a low multiplicity of infection.Semantic proximity Read ↗

Platelet gene therapy corrects the hemophilic phenotype in immunocompromised hemophilia A mice transplanted with genetically manipulated human cord blood stem cells.Semantic proximity Read ↗

High-level transgene expression in induced pluripotent stem cell–derived megakaryocytes: correction of Glanzmann thrombastheniaSemantic proximity Read ↗

Platelet gene therapy by lentiviral gene delivery to hematopoietic stem cells restores hemostasis and induces humoral immune tolerance in FIX(null) mice.Semantic proximity Read ↗

Generation and rescue of a murine model of platelet dysfunction: the Bernard-Soulier syndrome.Semantic proximity Read ↗

Syngeneic transplantation of hematopoietic stem cells that are genetically modified to express factor VIII in platelets restores hemostasis to hemophilia A mice with preexisting FVIII immunity.Semantic proximity Read ↗

Amelioration of the macrothrombocytopenia associated with the murine Bernard-Soulier syndromeSemantic proximity Read ↗

In vivo enrichment of genetically manipulated platelets for murine hemophilia B gene therapy.Semantic proximity Read ↗

Long-term correction of hemophilia A mice following lentiviral mediated delivery of an optimized canine factor VIII gene.Semantic proximity Read ↗

Phenotypic correction of hemophilia A by ectopic expression of activated factor VII in platelets.Semantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.