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

Enhanced factor VIII heavy chain for gene therapy of hemophilia A.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Mar 2009

Chen Lingxia, Lu Hui, Wang Jinhui, Sarkar Rita, Yang Xiao, Wang Hongli, High Katherine A, Xiao Weidong

Abstract excerpt

Hemophilia A gene therapy using recombinant adenovirus-associated virus (AAV) vectors has been hampered by the size of the factor VIII (FVIII) cDNA. Previously, splitting the FVIII coding sequence into a heavy-chain (HC) fragment and a light-chain (LC) fragment for dual recombinant AAV vector delivery has been successfully explored. However, the main disadvantage of this approach is a "chain imbalance" problem in...

Read the complete abstract on PubMed

Topics

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

Share to Acids
Share to Adenoviridae

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Antigens

Share to Factor VIII

Share to Gene Expression Regulation

Share to Genetic Therapy

Share to Hemophilia A

Share to Mice, Knockout

Source

Open in PubMed Open full text

Identifiers

PMID: 19127250
DOI: 10.1038/mt.2008.292
PMCID: PMC2790722

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Enhanced factor VIII heavy chain for gene therapy of hemophilia A.PMID 19127250Read ↗

Engineered coagulation factor VIII with enhanced secretion and coagulation potential for hemophilia A gene therapySemantic proximity Read ↗

Hemophilia A gene therapy: current and next-generation approachesSemantic proximity Read ↗

Efficient Production of Dual Recombinant Adeno-Associated Viral Vectors for Factor VIII DeliverySemantic proximity Read ↗

Current and emerging gene therapies for haemophilia A and BSemantic proximity Read ↗

Bioengineered coagulation factor VIII enables long-term correction of murine hemophilia A following liver-directed adeno-associated viral vector deliverySemantic proximity Read ↗

Preclinical assessment of an optimized AAV-FVIII vector in mice and non-human primates for the treatment of hemophilia ASemantic proximity Read ↗

Gene therapy in an era of emerging treatment options for hemophilia B.Semantic proximity Read ↗

Characterization of Adeno-Associated Viral Vector-Mediated Human Factor VIII Gene Therapy in Hemophilia A MiceSemantic proximity Read ↗

Recombinant Adeno-Associated Viral Vectors Expressing Human Coagulation FIX-E456H Variant in Hemophilia B Mice.Semantic proximity Read ↗

Efficacy and safety of long-term prophylaxis in severe hemophilia A dogs following liver gene therapy using AAV vectors.Semantic proximity Read ↗

New approaches to gene and cell therapy for hemophilia.Semantic proximity Read ↗

Minimal Essential Human Factor VIII Alterations Enhance Secretion and Gene Therapy EfficiencySemantic proximity Read ↗

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