Basic Information

Gene symbol ATP2A2 Synonyms ATP2B, DAR, DD, SERCA2 Type of gene protein-coding
Description ATPase sarcoplasmic/endoplasmic reticulum Ca2+ transporting 2

GTO ID GTC0043
Trial ID NCT00534703
Disease Heart Failure
Altered gene SERCA2
Therapeutic/Target gene Therapeutic gene
TherapyGene transfer
Treatment AAV1-SERCA2a
PhasePhase2
Recruitment statusTerminated
TitleInvestigation of the Safety and Feasibility of AAV1/SERCA2a Gene Transfer in Patients With Chronic Heart Failure and a Left Ventricular Assist Device
Year2007
CountryUnited Kingdom
Company sponsorImperial College London
Other ID(s)CRO782|2007-002809-48
Vector information
Vectoradeno-associated virus
ConstructAAV1-CMV-SERCA2a
Vector typeadeno-associated virus serotype 1 (AAV1) vector
Transgene/Inserted genesarcoplasmic reticulum calcium ATPase 2a cDNA
Regulatory elementcytomegalovirus (CMV) promoter

Clinical Result

Cohort 1
Administration route delivered by a percutaneous method in the catheter laboratory
Dosage 1E13 DRP (DNase resistant particles)
Pts 4
Age Adult, Older_Adult
Adverse reactions 1/4(All-cause mortality); 2/4(Cardiac disorders; General disorders)
References PMID: 32669717

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