Recombinant AAV2-mediated β-globin expression in human fetal hematopoietic cells from the aborted fetuses with β-thalassemia major

scientific article published on 27 May 2011

Recombinant AAV2-mediated β-globin expression in human fetal hematopoietic cells from the aborted fetuses with β-thalassemia major is …
instance of (P31):
scholarly articleQ13442814

External links are
P356DOI10.1007/S12185-011-0823-X
P698PubMed publication ID21617888

P2093author name stringJing Tian
Feng Wang
Fei Zhao
Jin-Feng Xue
Meng-Qun Tan
Liu-Jiang Song
P2860cites workIsolation of highly infectious and pure adeno-associated virus type 2 vectors with a single-step gravity-flow column.Q31934499
High-level beta-globin expression after retroviral transfer of locus activation region-containing human beta-globin gene derivatives into murine erythroleukemia cellsQ33560915
Recruitment of single-stranded recombinant adeno-associated virus vector genomes and intermolecular recombination are responsible for stable transduction of liver in vivoQ33811409
Recombinant AAV2 transduction of primitive human hematopoietic stem cells capable of serial engraftment in immune-deficient miceQ33911508
AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B.Q34168942
Phenotype-genotype relationships in monogenic disease: lessons from the thalassaemias.Q34205528
Assessing the potential for AAV vector genotoxicity in a murine modelQ34754355
American Society of Gene Therapy (ASGT) ad hoc subcommittee on retroviral-mediated gene transfer to hematopoietic stem cellsQ35195407
Use of Adeno-Associated Virus as a General Transduction Vector for Mammalian CellsQ35228018
Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vectorQ35872651
Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapyQ35916142
Existence of transient functional double-stranded DNA intermediates during recombinant AAV transductionQ35928797
Hematopoietic stem cell transduction by recombinant adeno-associated virus vectors: problems and solutionsQ36184772
Key factors in experimental mouse hematopoietic stem cell transplantationQ36547328
Adeno-associated virus-mediated gene transfer in hematopoietic stem/ progenitor cells as a therapeutic toolQ36683381
Gene therapy clinical trials worldwide to 2007--an update.Q36920192
Current status of globin gene therapy for the treatment of beta-thalassaemia.Q37137998
High-level beta-globin expression and preferred intragenic integration after lentiviral transduction of human cord blood stem cellsQ40051271
Impaired nuclear transport and uncoating limit recombinant adeno-associated virus 2 vector-mediated transduction of primary murine hematopoietic cells.Q40463241
Genetic treatment of severe hemoglobinopathies: the combat against transgene variegation and transgene silencing.Q41752960
Isolation and therapeutic potential of human haemopoietic stem cellsQ41787137
Lineage-specific expression of a human beta-globin gene in murine bone marrow transplant recipients reconstituted with retrovirus-transduced stem cellsQ41904037
Adeno-associated virus 2-mediated transduction and erythroid lineage-restricted long-term expression of the human beta-globin gene in hematopoietic cells from homozygous beta-thalassemic miceQ43641175
Successful correction of the human beta-thalassemia major phenotype using a lentiviral vectorQ45005788
Efficient gene transfer into human cord blood CD34+ cells and the CD34+CD38- subset using highly purified recombinant adeno-associated viral vector preparations that are free of helper virus and wild-type AAV.Q45151455
Phase I trial of intramuscular injection of a recombinant adeno-associated virus alpha 1-antitrypsin (rAAV2-CB-hAAT) gene vector to AAT-deficient adultsQ45676046
Transfer of contaminants in adeno-associated virus vector stocks can mimic transduction and lead to artifactual resultsQ45759275
AAV2-mediated ocular gene therapy for infantile neuronal ceroid lipofuscinosis.Q45884511
A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies.Q51708365
Identification and characterization of three classes of erythroid progenitors in human fetal liverQ70864249
P433issue6
P304page(s)691-699
P577publication date2011-05-27
P1433published inInternational Journal of HematologyQ6051416
P1476titleRecombinant AAV2-mediated β-globin expression in human fetal hematopoietic cells from the aborted fetuses with β-thalassemia major
P478volume93

Reverse relations

Q37011792Optimizing the transduction efficiency of capsid-modified AAV6 serotype vectors in primary human hematopoietic stem cells in vitro and in a xenograft mouse model in vivo.cites workP2860