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SCTi003-A-3

Registration Summary:
SCTi003-A-3

General

Cell Line
hPSCreg name SCTi003-A-3
Cite as:
SCTi003-A-3
Cell line type Human induced pluripotent stem cell (hiPSC)
Similar lines No similar lines found.
Last update 26th August 2025
Notes Genome-edited Human iPSC Line, SCTi003-A-3, ABCA4 Knockout (SCTi003-A-3) was generated by CRISPR/Cas9 technology, which created a loss-of-function (knockout) mutation in the ATP-binding cassette sub-family A member 4 (ABCA4) gene. ABCA4 is associated with inherited retinal disorders such as Stargardt disease, cone-rod dystrophy, and retinitis pigmentosa.

This product’s parental human induced pluripotent stem cell (hiPSC) line, Healthy Control Human hiPSC Line, Female, SCTi003-A, is a well-characterized control line derived from peripheral blood mononuclear cells (PBMCs) from a 48-year-old donor. SCTi003-A-3 has been validated by sequencing to confirm frameshift-inducing indels resulting in the complete loss of ABCA4 protein expression. Post-editing, extensive quality control procedures were undertaken in the manufacturing process for SCTi003-A-3 to ensure optimal product performance and reproducibility. SCTi003-A-3 is karyotypically stable, expresses markers of the undifferentiated state, and remains capable of directed differentiation into all three germ layers, including retinal lineage cells relevant for retinal disease research and therapeutic development. This genome-edited hiPSC line, along with its parental hiPSC line, enables precise investigation of ABCA4-linked disease mechanisms and functional genomics, as well as high-throughput screening of therapeutics targeting retinal degeneration.

SCTi003-A-3 is manufactured with mTeSR™ Plus (Catalog #100-0276) and is compatible with STEMdiff™ cell culture media products, allowing for standardized high-quality maintenance and differentiation to various cell types, including retinal pigment epithelium cells.

Cells were obtained using Institutional Review Board (IRB)-approved consent forms and protocols.
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Provider
Generator STEMCELL Technologies Inc. (SCT)
Owner STEMCELL Technologies Inc. (SCT)
Distributors
Derivation country United States

External Databases
BioSamples SAMEA119586896

General Information
* Is the cell line readily obtainable for third parties?
Yes
Research use: allowed
Clinical use: not allowed
Commercial use: allowed
Subclone of

Donor Information

General Donor Information
Sex female
Ethnicity Self-declared race/ethnicity = White
Ancestry = 100% European

Phenotype and Disease related information (Donor)
Diseases No disease was diagnosed.
Is the medical history available upon request? No
Is clinical information available? No

Other Genotyping (Donor)
Is there genome-wide genotyping or functional data available?
No

External Databases (Donor)
BioSamples SAMEA11371632

Ethics

Also have a look at the ethics information for the parental line SCTi003-A .
Is there an MTA available for the cell line? No
For generation of the cell line, who was the supplier of any recombined DNA vectors or commercial kits used?
Are you aware of any constraints on the use or distribution of the cell line from the owner or any parties identified in the query above? No

hIPSC Derivation

General
The source cell information can be found in the parental cell line SCTi003-A.

Reprogramming method
Vector type Non-integrating
Vector Proprietary non-integrating reprogramming technology
Is reprogramming vector detectable?
No
Methods used
PCR
Notes on reprogramming vector detection Clearance confirmed at passage 21

Vector free reprogramming
Type of used vector free reprogramming factor(s)
None

Other
Derived under xeno-free conditions
No
Derived under GMP?
No
Available as clinical grade?
No

Culture Conditions

Surface coating Matrigel/Geltrex
Feeder cells
No
Passage method Enzyme-free cell dissociation
ReLeSR™
O2 Concentration 20 %
CO2 Concentration 5 %
Medium mTeSR™ Plus
Has Rock inhibitor (Y27632) been used at passage previously with this cell line?
Yes
Has Rock inhibitor (Y27632) been used at cryo previously with this cell line?
No
Has Rock inhibitor (Y27632) been used at thaw previously with this cell line?
No

Characterisation

Analysis of Undifferentiated Cells
Marker Expressed Immunostaining RT-PCR Flow Cytometry Enzymatic Assay Expression Profiles
POU5F1 (OCT-4)
Yes
–
TRA 1-60
Yes
–
Score:
Marker Present Absent
mCpG
OCT4
Morphology pictures
Figure 2. SCTi003-A-3 Human iPSCs Demonstrate High-Quality Morphology in Routine Culture.

Cryopreserved cells from line SCTi003-A-3 were thawed and maintained in mTeSR™ Plus (Catalog #100-1130) on Corning® Matrigel® Matrix. (A) The resulting iPSC colonies have densely packed cells and show multi-layering when ready to be passaged. (B,C) Cells retain prominent nucleoli and high nuclear-to-cytoplasmic ratios. iPSC = induced pluripotent stem cell.
Differentiation Potency
Endoderm
Ont Id: UBERON_0000925
In vitro directed differentiation
Marker Expressed
SOX17
Yes
CXCR4
Yes
Protocol or reference
STEMdiff Trilineage Kit PIS.pdf
Mesoderm
Ont Id: UBERON_0000926
In vitro directed differentiation
Marker Expressed
TBXT
Yes
NCAM1
Yes
Protocol or reference
STEMdiff Trilineage Kit PIS.pdf
Ectoderm
Ont Id: UBERON_0000924
In vitro directed differentiation
Marker Expressed
PAX6
Yes
NES
Yes
Protocol or reference
STEMdiff Trilineage Kit PIS.pdf

Microbiology / Virus Screening
HIV 1 Negative
HIV 2 Negative
Hepatitis B Negative
Hepatitis C Negative
Mycoplasma Negative

Certificate of Analysis
Is there a certificate of analysis available?
Yes
SCTi003-A-3_Lot_2505412010_COA.pdf
Passage: 37

Genotyping

Karyotyping (Cell Line)
Has the cell line karyotype been analysed?
Yes
46,XX
SCTi003-A-3_Karyotype_(CCB).tif
Passage number: 37
Karyotyping method: G-Banding

Other Genotyping (Cell Line)
Is there genome-wide genotyping or functional data available?
Yes
Exome sequencing
Whole exome sequencing data file (Catalog #500-0709) is available upon request for a fee for SCTi003-A-3 customers. Please contact iPSCrequests@stemcell.com for more information.
SNP typing array
SNP microarray data is included in each lot-specific COA. Please contact iPSCrequests@stemcell.com for more information.
Genome Sequencing
Whole genome sequencing data file (Catalog #500-0730) is available upon request for a fee for SCTi003-A-3 customers. Please contact iPSCrequests@stemcell.com for more information.

Genetic Modification

Disease/phenotype related modifications
Stargardt Disease
Synonyms
  • Stargardt Disease
Genetic modifications
ABCA4 - ATP binding cassette subfamily A member 4 (target)
Gene knock-out
1p22.1
The SCTi003-A-3 iPSC line was derived from the parental SCTi003-A line, which originated from a healthy female donor with a wild-type ABCA4 genotype. Using CRISPR-Cas9 genome editing, two independent frameshift mutations were introduced in exon 3 of the ABCA4 gene (NM_000350.3) to generate a biallelic knockout.

On allele 1, a single-base duplication at c.209dupT changes the reading frame beginning at codon 71, producing a frameshift mutation (p.Gln71fs). On allele 2, a single-base deletion at c.207delG alters the reading frame beginning at codon 69, resulting in a second frameshift mutation (p.Trp69fs).

Both frameshift mutations are present in trans, resulting in a compound heterozygous ABCA4 knockout. Each mutation is predicted to disrupt ABCA4 function, and together they result in loss of functional ABCA4 protein. This engineered model provides a genetically defined system for investigating the role of ABCA4 loss-of-function in retinal biology and for evaluating candidate therapeutic compounds.
CRISPR-associated (CRISPR/Cas) System