Target intelligence / Profile preview

Albumin genomic locus (ALB locus)

Target
ALB locus
Molecular classification
Genomic locus, Other
01

Overview

The Albumin genomic locus (ALB) is the chromosomal region on 4q13.3 responsible for encoding albumin, the most abundant protein in human plasma (UniProt P02768). In the field of genetic medicine, this locus is utilized as a 'safe harbor' for in vivo gene editing because of its exceptionally high transcriptional activity in hepatocytes (Sangamo Therapeutics, 2024). By inserting a therapeutic transgene into the first intron of the albumin gene using tools like Zinc Finger Nucleases (ZFNs), researchers can leverage the powerful endogenous albumin promoter to drive the secretion of high levels of therapeutic proteins into the bloodstream (PubMed: 29107332). This 'bio-factory' approach is currently being investigated for the treatment of monogenic disorders such as Hemophilia B and various Lysosomal Storage Diseases (NIH ClinicalTrials.gov). While the primary biological role of the locus is maintaining systemic osmotic pressure and transporting hormones and fatty acids, its therapeutic value lies in providing a stable, high-output platform for protein replacement therapy.

Other names
ALB geneSerum albumin locusAlbumin safe harbor4q13.3 locus
02

Mechanism of action

Targeted gene integration via genome editing (e.g., Zinc Finger Nucleases) to utilize the endogenous albumin promoter for high-level expression of therapeutic proteins.

03

Biological functions

Protein synthesisRegulation of plasma oncotic pressureLigand transportMaintenance of blood pH
04

Disease associations

Hemophilia BMucopolysaccharidosis type IMucopolysaccharidosis type IIAnalbuminemiaFabry disease
05

Safety considerations

Off-target genomic cleavageInsertional mutagenesisImmune response to Adeno-associated virus (AAV) vectorsPotential reduction in endogenous albumin productionGenotoxicity
06

Interacting drugs

SB-FIX

3 more in the full profile.

07

Biomarkers

Serum albumin levelsCirculating therapeutic protein levels (e.g., Factor IX, IDS, IDUA)Vector DNA copy numberLiver transaminases (ALT/AST)

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