Basic Information

Gene symbol CCR5 Synonyms CC-CKR-5, CCCKR5, CCR-5, CD195, CKR-5, CKR5, CMKBR5, IDDM22 Type of gene protein-coding
Gene perturbation-related omics dataset PertOrg
Description C-C motif chemokine receptor 5

Gene symbol tat Synonyms None Type of gene protein-coding
Description Tat

Gene symbol vif Synonyms None Type of gene protein-coding
Description Vif

GTO ID GTC0382
Trial ID NCT03215004
Disease HIV Infection
Altered gene CCR5|tat|vif
Therapeutic/Target gene Target gene
TherapyGene transfer
Treatment AGT103-T
Recruitment statusUnknown
TitleDeveloping a Functional Cure for HIV Disease: Clinical Specimen Collection From HIV Positive Individuals
Year2017
CountryUnited States
Company sponsorAmerican Gene Technologies International Inc.
Other ID(s)AGT-CS168
Vector information
Vectorlentivirus
Vector typethe third generation, self-inactivating lentivirus vector
Transgene/Inserted geneA cluster of three engineered microRNAs (miRNAs) designed to decrease RNA levels through the recognition of target sequences in mRNA encoding the host protein CCR5, as well as in genomic or sub-genomic RNA sequences from the HIV Tat and Vif genes.
Regulatory elementEF1α promoter; a Woodchuck hepatitis virus post-transcriptional regulatory element (WPRE)

Clinical Result

Cohort 1
Administration route infusion
Dosage 2E9~2.1E10 cells/kg of body weight
Donor type autologous
Pts 24
Age Adult
Lymph depletion Yes
Outcome Gag-specific CD4+ T cells were detected in all treated individuals and were substantially increased by 9 to 300-fold compared to baseline, by 14 days after cell product infusion. Gag-specific CD8+ T cells were increased by 1.7 to 10-fold relative to baseline, by 28 days after cell product infusion. Levels of Gag-specific CD4+ T cells remained high (~2 to 70-fold higher relative to baseline) throughout 3-6 months after infusion.
Adverse reactions No serious clinical adverse events
References PMID: 36452901

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