SLC66A1

Summary

SLC66A1 (solute carrier family 66 member 1, HGNC:26001) is a protein-coding gene on chromosome 1p36.13, encoding Lysosomal amino acid transporter 1 homolog (Q6ZP29). Amino acid transporter that specifically mediates the pH-dependent export of the cationic amino acids arginine, histidine and lysine from lysosomes.

Enables L-arginine transmembrane transporter activity; L-histidine transmembrane transporter activity; and L-lysine transmembrane transporter activity. Involved in L-amino acid transport and intracellular amino acid homeostasis. Located in lysosomal membrane.

Source: NCBI Gene 54896 — RefSeq curated summary.

At a glance

• Gene–disease (curated): retinitis pigmentosa (Strong, GenCC)
• GWAS associations: 3
• Clinical variants (ClinVar): 79 total — 1 pathogenic
• MANE Select transcript: NM_001040125

Identifiers

Gene identifiers

Field	Value
HGNC ID	HGNC:26001
Approved symbol	SLC66A1
Name	solute carrier family 66 member 1
Location	1p36.13
Locus type	gene with protein product
Status	Approved
Aliases	FLJ20320, LAAT-1, LAAT1
Ensembl gene	ENSG00000040487
Ensembl biotype	protein_coding
OMIM	614760
Entrez	54896

Gene structure

Transcript identifiers

Ensembl transcripts: 10 — 8 protein_coding, 2 nonsense_mediated_decay

ENST00000375153, ENST00000375155, ENST00000400548, ENST00000432465, ENST00000469076, ENST00000497827, ENST00000902231, ENST00000902232, ENST00000956590, ENST00000956591

RefSeq mRNA: 4 — MANE Select: NM_001040125 NM_001040125, NM_001040126, NM_001287531, NM_017765

CCDS: CCDS195, CCDS30618

Canonical transcript exons

ENST00000375153 — 8 exons

Exon	Start	End
ENSE00000754876	19325495	19325582
ENSE00001465925	19312326	19312889
ENSE00001889561	19328572	19329300
ENSE00002267461	19317600	19317841
ENSE00003489187	19326245	19326387
ENSE00003490822	19324633	19324762
ENSE00003576994	19326531	19326623
ENSE00003625916	19327227	19327412

Expression profiles

Bgee: expression breadth ubiquitous, 172 present calls, max score 90.82.

FANTOM5 (CAGE): breadth ubiquitous, TPM avg 16.4860 / max 191.1711, expressed in 1807 samples.

FANTOM5 promoters (2 alternative TSS)

Promoter ID	TPM avg	Samples expressed
1060	16.2390	1807
1061	0.2469	92

Top tissues by expression

234 total, by Bgee expression score (0-100, higher = more expressed):

Tissue	Anatomy ID	Expression score	Quality
right hemisphere of cerebellum	UBERON:0014890	90.82	gold quality
cerebellar hemisphere	UBERON:0002245	90.53	gold quality
cerebellar cortex	UBERON:0002129	90.36	gold quality
right adrenal gland	UBERON:0001233	89.41	gold quality
right adrenal gland cortex	UBERON:0035827	88.90	gold quality
adenohypophysis	UBERON:0002196	88.83	gold quality
left adrenal gland	UBERON:0001234	88.82	gold quality
left adrenal gland cortex	UBERON:0035825	88.74	gold quality
cerebellum	UBERON:0002037	88.43	gold quality
right lobe of liver	UBERON:0001114	88.27	gold quality
adrenal cortex	UBERON:0001235	87.19	gold quality
pituitary gland	UBERON:0000007	86.99	gold quality
adrenal gland	UBERON:0002369	86.47	gold quality
apex of heart	UBERON:0002098	86.39	gold quality
small intestine Peyer’s patch	UBERON:0003454	86.02	gold quality
body of stomach	UBERON:0001161	85.69	gold quality
C1 segment of cervical spinal cord	UBERON:0006469	84.89	gold quality
mucosa of transverse colon	UBERON:0004991	84.87	gold quality
left testis	UBERON:0004533	84.85	gold quality
metanephros cortex	UBERON:0010533	84.82	gold quality
primordial germ cell in gonad	CL:0000670 ∩ UBERON:0000991	84.71	gold quality
right testis	UBERON:0004534	84.67	gold quality
right lobe of thyroid gland	UBERON:0001119	84.62	gold quality
body of pancreas	UBERON:0001150	84.48	gold quality
right frontal lobe	UBERON:0002810	84.29	gold quality
gastrocnemius	UBERON:0001388	84.27	gold quality
lower esophagus mucosa	UBERON:0035834	83.93	gold quality
granulocyte	CL:0000094	83.90	gold quality
left lobe of thyroid gland	UBERON:0001120	83.58	gold quality
endocervix	UBERON:0000458	83.42	gold quality

Single-cell (SCXA)

Detected in 1 experiment(s), a significant marker in 1.

Experiment	Marker?	Max mean expression
E-ANND-3	yes	4.05

Regulation

Is transcription factor: no

miRNA regulators (miRDB)

21 targeting SLC66A1, top 30 by miRDB confidence (max_score; target_count = how many genes the miRNA targets in total — lower means more specific):

miRNA	Max score	Avg score	miRNA target_count
HSA-MIR-4500	99.99	72.72	2367
HSA-LET-7A-5P	99.98	72.29	1790
HSA-LET-7B-5P	99.98	72.31	1790
HSA-LET-7C-5P	99.98	72.29	1790
HSA-LET-7E-5P	99.98	72.29	1790
HSA-LET-7F-5P	99.98	72.56	1784
HSA-LET-7G-5P	99.98	72.37	1784
HSA-LET-7I-5P	99.98	72.37	1788
HSA-MIR-98-5P	99.98	72.33	1787
HSA-MIR-6778-3P	99.96	67.29	2693
HSA-MIR-6825-5P	99.96	69.81	3431
HSA-MIR-1976	99.74	65.48	1127
HSA-MIR-6852-5P	99.17	66.69	2073
HSA-MIR-1910-3P	98.44	67.51	1695
HSA-MIR-6511A-5P	98.13	67.47	1770
HSA-MIR-6747-3P	97.73	64.84	1596
HSA-MIR-663B	97.40	62.91	664
HSA-MIR-101-2-5P	95.96	68.62	55
HSA-MIR-1238-3P	95.27	62.25	552
HSA-MIR-1231	95.10	65.63	663
HSA-MIR-6879-3P	93.93	64.00	759

Literature-anchored findings (GeneRIF, showing 3)

• PQLC2 and Ypq1-3 proteins are lysosomal/vacuolar exporters of CAAs and suggest that small-molecule transport is a conserved feature of the PQ-loop protein family (PMID:23169667)
• knockdown caused growth arrest and cell death of cancer cells and suppressed tumor growth in a mouse xenograft model. These results suggest that targeting PQLC2 is an effective strategy for GC treatment. (PMID:30729615)
• Receptor-like role for PQLC2 amino acid transporter in the lysosomal sensing of cationic amino acids. (PMID:33597295)

Cross-species orthologs

4 orthologs

Organism	Symbol	Gene ID
danio_rerio	slc66a1	ENSDARG00000043624
mus_musculus	Slc66a1	ENSMUSG00000028744
rattus_norvegicus	Slc66a1	ENSRNOG00000017706
caenorhabditis_elegans		WBGENE00021546

Paralogs (1): TMEM44 (ENSG00000145014)

Protein

Protein identifiers

Lysosomal amino acid transporter 1 homolog — Q6ZP29 (reviewed: Q6ZP29)

Alternative names: PQ-loop repeat-containing protein 2, Solute carrier family 66 member 1

All UniProt accessions (5): A0A024RAA1, A2A2E6, E9PJZ6, E9PL93, Q6ZP29

UniProt curated annotations — full annotation on UniProt →

Function. Amino acid transporter that specifically mediates the pH-dependent export of the cationic amino acids arginine, histidine and lysine from lysosomes.

Subcellular location. Lysosome membrane.

Domain organisation. The di-leucine motif mediates lysosomal localization.

Miscellaneous. May play a role in the export from lysosomes of cysteamine-cysteine mixed disulfide (MxD), the product formed upon treatment by cysteamine of patients with cystinosis, a disease characterized by the accumulation of cystine in the lysosomes.

Similarity. Belongs to the laat-1 family.

Isoforms (3)

UniProt ID	Names	Canonical?
Q6ZP29-1	1	yes
Q6ZP29-2	2
Q6ZP29-3	3

RefSeq proteins (4): NP_001035214, NP_001035215, NP_001274460, NP_060235 (=MANE)

Domains & families (InterPro)

ID	Name	Type
IPR006603	PQ-loop_rpt	Repeat
IPR051415	LAAT-1	Family

Pfam: PF04193

UniProt features (29 total): topological domain 8, transmembrane region 7, sequence conflict 4, domain 2, splice variant 2, mutagenesis site 2, chain 1, short sequence motif 1, glycosylation site 1, sequence variant 1

Structure

Experimental structures (PDB)

0 structures.

Predicted structure (AlphaFold)

Model	pLDDT	Fraction very-high
AF-Q6ZP29-F1	83.40	0.47

Functional residue map

Curated UniProt residues grouped by drug-discovery relevance — catalytic, ligand-binding, modification, and mutation-validated positions. Source: UniProtKB sequence features.

Glycosylation sites (1): 10

Mutagenesis-validated functional residues (2):

Position	Phenotype
55	abolishes uptake of arginine and lysine.
288–289	abolishes lysosomal localization.

Function

Pathways and Gene Ontology

Reactome pathways

2 pathways

ID	Pathway
R-HSA-5223345	Miscellaneous transport and binding events
R-HSA-382551	Transport of small molecules

MSigDB gene sets: 115 (showing top): GOCC_VACUOLAR_MEMBRANE, GOBP_AMINO_ACID_TRANSMEMBRANE_TRANSPORT, CAGCTG_AP4_Q5, GOBP_ORGANIC_ACID_TRANSPORT, GOBP_AMINO_ACID_TRANSPORT, GOBP_BASIC_AMINO_ACID_TRANSPORT, GOBP_ORGANIC_ANION_TRANSPORT, LASTOWSKA_NEUROBLASTOMA_COPY_NUMBER_DN, GOBP_ORGANIC_CATION_TRANSPORT, GOBP_TRANSMEMBRANE_TRANSPORT, GOBP_AZOLE_TRANSMEMBRANE_TRANSPORT, GOBP_HOMEOSTATIC_PROCESS, GOBP_CHEMICAL_HOMEOSTASIS, GOMF_L_AMINO_ACID_TRANSMEMBRANE_TRANSPORTER_ACTIVITY, GOMF_AMINO_ACID_TRANSMEMBRANE_TRANSPORTER_ACTIVITY

GO Biological Process (7): lysine transport (GO:0015819), transmembrane transport (GO:0055085), intracellular amino acid homeostasis (GO:0080144), L-histidine transmembrane transport (GO:0089709), L-lysine transmembrane transport (GO:1903401), L-arginine transmembrane transport (GO:1903826), amino acid transport (GO:0006865)

GO Molecular Function (5): L-histidine transmembrane transporter activity (GO:0005290), basic amino acid transmembrane transporter activity (GO:0015174), L-lysine transmembrane transporter activity (GO:0015189), L-arginine transmembrane transporter activity (GO:0061459), protein binding (GO:0005515)

GO Cellular Component (4): lysosomal membrane (GO:0005765), organelle membrane (GO:0031090), lysosome (GO:0005764), membrane (GO:0016020)

Reactome top-level categories

Rollup of top-1 pathways:

Category	Pathways
Transport of small molecules	1

GO top-level categories

Rollup of top GO terms by namespace:

Category	Terms
L-alpha-amino acid transmembrane transport	3
basic amino acid transmembrane transporter activity	3
L-amino acid transmembrane transporter activity	3
L-lysine transport	2
transport	2
basic amino acid transmembrane transport	2
basic amino acid transport	1
cellular process	1
intracellular chemical homeostasis	1
aromatic amino acid transport	1
azole transmembrane transport	1
aromatic amino acid transmembrane transporter activity	1
L-histidine transmembrane transport	1
azole transmembrane transporter activity	1
amino acid transmembrane transporter activity	1
L-lysine transmembrane transport	1
L-arginine transmembrane transport	1
binding	1
lysosome	1
lytic vacuole membrane	1
membrane	1
membrane-bounded organelle	1
lytic vacuole	1
cellular anatomical structure	1

Protein interactions and networks

STRING

548 interactions, top by confidence (×1000):

Protein A	Protein B	Partner UniProt	Score
SLC66A1	CTNS	O60931	811
SLC66A1	C9orf72	Q96LT7	575
SLC66A1	SLC36A1	Q7Z2H8	565
SLC66A1	SLC38A7	Q9NVC3	565
SLC66A1	WDR41	Q9HAD4	546
SLC66A1	SMCR8	Q8TEV9	544
SLC66A1	SLC38A9	Q8NBW4	539
SLC66A1	ZSWIM2	Q8NEG5	451
SLC66A1	NPRL3	Q12980	451
SLC66A1	MRTO4	Q9UKD2	433
SLC66A1	EBNA1BP2	Q99848	421
SLC66A1	ELP2	Q6IA86	396
SLC66A1	MCOLN1	Q9GZU1	389
SLC66A1	TMEM175	Q9BSA9	377
SLC66A1	TASOR2	Q5VWN6	360

IntAct

1 interactions, top by confidence:

BioGRID (13): PQLC2 (Affinity Capture-RNA), PQLC2 (Two-hybrid), SLC35C2 (Two-hybrid), AQP6 (Two-hybrid), GPRC5D (Two-hybrid), GJA8 (Two-hybrid), SLC39A2 (Two-hybrid), NINJ2 (Two-hybrid), GPR152 (Two-hybrid), GJC1 (Two-hybrid), GJA5 (Two-hybrid), PQLC2 (Negative Genetic), PQLC2 (Affinity Capture-RNA)

ESM2 similar proteins: A1L272, A4FV75, A5A6S6, A6QL92, B0BMY1, B8AF63, F1NXU8, O54902, O80605, P49281, P49282, P57057, P57758, Q17QZ3, Q28HR4, Q3TIT8, Q5EAL3, Q5F383, Q5F3N0, Q5R6J3, Q5R839, Q5RD30, Q5ZJX0, Q640L2, Q6DHU1, Q6DIV6, Q6IR74, Q6J4K2, Q6NTJ7, Q6P499, Q6PF45, Q6YK44, Q6ZP29, Q8BGN5, Q8BJA2, Q8BXA5, Q8C4N4, Q8IVJ1, Q8NA29, Q8NCC5

Diamond homologs: A1A4F0, B0BMY1, Q5ZJX0, Q6ZP29, Q8C4N4, Q95XZ6, Q10482, Q5M880, Q80XM9, Q9VCR7

SIGNOR signaling

0 interactions.

Disease & clinical

Clinical variants and AI predictions

ClinVar

79 variants total. Per-class counts are floors (≥ shown; pagination cap):

Classification	Count (floor)
Pathogenic	1
Likely pathogenic	0
Uncertain significance	56
Likely benign	3
Benign	0

Top pathogenic / likely-pathogenic (1)

Variant ID	HGVS	Classification
4751936	NM_001040125.2(SLC66A1):c.618+1G>A	Pathogenic

SpliceAI

1568 predictions. Top by Δscore:

Variant	Effect	Δscore
1:19324630:C:G	acceptor_gain	1.0000
1:19324630:CA:C	acceptor_loss	1.0000
1:19324631:A:AG	acceptor_gain	1.0000
1:19324631:A:T	acceptor_loss	1.0000
1:19324632:G:GG	acceptor_gain	1.0000
1:19324632:GCC:G	acceptor_gain	1.0000
1:19324632:GCCA:G	acceptor_gain	1.0000
1:19324760:CAG:C	donor_loss	1.0000
1:19324761:AGGTG:A	donor_loss	1.0000
1:19324762:GGTG:G	donor_loss	1.0000
1:19324764:T:G	donor_loss	1.0000
1:19325578:TCTGT:T	donor_gain	1.0000
1:19325581:GT:G	donor_gain	1.0000
1:19325582:TGTG:T	donor_loss	1.0000
1:19325583:G:GG	donor_gain	1.0000
1:19325584:T:G	donor_loss	1.0000
1:19325585:GAGT:G	donor_loss	1.0000
1:19325586:AGTA:A	donor_loss	1.0000
1:19326240:TGCA:T	acceptor_loss	1.0000
1:19326241:GCAGT:G	acceptor_loss	1.0000
1:19326242:CAGTG:C	acceptor_loss	1.0000
1:19326243:A:AG	acceptor_gain	1.0000
1:19326244:G:GT	acceptor_gain	1.0000
1:19326244:GT:G	acceptor_gain	1.0000
1:19326244:GTGT:G	acceptor_gain	1.0000
1:19326244:GTGTC:G	acceptor_gain	1.0000
1:19326529:A:AG	acceptor_gain	1.0000
1:19326530:G:GG	acceptor_gain	1.0000
1:19326530:GC:G	acceptor_gain	1.0000
1:19326530:GCC:G	acceptor_gain	1.0000

AlphaMissense

1865 scored. Top likely-pathogenic:

Variant	Protein change	am_pathogenicity
1:19317822:T:C	F49L	0.998
1:19317824:T:A	F49L	0.998
1:19317824:T:G	F49L	0.998
1:19324700:T:A	W78R	0.998
1:19324700:T:C	W78R	0.998
1:19326579:A:C	S192R	0.998
1:19326581:C:A	S192R	0.998
1:19326581:C:G	S192R	0.998
1:19327289:C:A	N227K	0.998
1:19327289:C:G	N227K	0.998
1:19327383:A:C	S259R	0.998
1:19327385:C:A	S259R	0.998
1:19327385:C:G	S259R	0.998
1:19327405:A:C	D266A	0.998
1:19324713:A:C	D82A	0.997
1:19326568:G:A	G188D	0.997
1:19327371:T:A	W255R	0.997
1:19327371:T:C	W255R	0.997
1:19327404:G:C	D266H	0.997
1:19327405:A:T	D266V	0.997
1:19317819:T:C	C48R	0.996
1:19324712:G:C	D82H	0.996
1:19324713:A:T	D82V	0.996
1:19324723:C:A	N85K	0.996
1:19324723:C:G	N85K	0.996
1:19327284:G:T	G226W	0.996
1:19324688:T:C	F74L	0.995
1:19324689:T:C	F74S	0.995
1:19324690:C:A	F74L	0.995
1:19324690:C:G	F74L	0.995

dbSNP variants (sampled 300 via entrez): RS1000061803 (1:19319806 G>A), RS1000083657 (1:19310817 T>G), RS1000167266 (1:19315551 A>G), RS1000265913 (1:19325643 G>A,C,T), RS1000439086 (1:19320630 T>A,C,G), RS1000444243 (1:19310455 T>C), RS1000531598 (1:19321101 G>A,T), RS1000538528 (1:19311456 G>A,C), RS1000599480 (1:19316530 A>G), RS1000739254 (1:19324994 AAGG>A), RS1000975808 (1:19329896 T>G), RS1001005319 (1:19324840 C>T), RS1001119566 (1:19321826 A>C,G,T), RS1001182856 (1:19311668 T>C,G), RS1001183460 (1:19322131 G>A,T)

Disease associations

OMIM: gene MIM:614760 | disease phenotypes: MIM:219800

GenCC curated gene-disease

Disease	Classification	Inheritance
retinitis pigmentosa	Strong	Autosomal recessive

Mondo (2): nephropathic cystinosis (MONDO:0100151), retinitis pigmentosa (MONDO:0019200)

Orphanet (2): Cystinosis (Orphanet:213), Infantile nephropathic cystinosis (Orphanet:411629)

HPO phenotypes

0 total (0 of 0 shown, HPO-id order):

GWAS associations

3 associations (top):

Study	Trait	p-value
GCST007001_1	Cerebrospinal AB1-42 levels in normal cognition	5.000000e-07
GCST007932_49	Medication use (thyroid preparations)	9.000000e-09
GCST008758_46	Pre-treatment viral load in HIV-1 infection	2.000000e-17

EFO canonical traits (3, from GWAS)

EFO ID	Trait name
EFO:0004670	beta-amyloid 1-42 measurement
EFO:0009933	Thyroid preparation use measurement
EFO:0010125	viral load

MeSH disease descriptors (2)

Descriptor	Name	Tree numbers
D012174	Retinitis Pigmentosa	C11.270.684; C11.768.585.658.500; C16.320.290.684
C535335	Abderhalden-Kaufmann-Lignac syndrome (supp.)

Drugs & pharmacology

Drug and pharmacology data

Is drug target: no

PharmGKB: 1 entry (VIP=true, CPIC=false)

GtoPdb / IUPHAR curated pharmacology

(IUPHAR/BPS Guide to Pharmacology — expert-curated)

Target class: transporter — SLC66 Lysosomal amino acid transporters

CTD chemical–gene interactions

24 total (human), top 24 by PubMed support.

Chemical	Actions (top 5)	PubMed papers
Acetaminophen	increases expression	3
Cyclosporine	increases expression	3
sodium arsenite	increases expression, affects cotreatment, increases abundance	2
Arsenic	affects methylation, affects cotreatment, increases abundance, increases expression	2
Valproic Acid	affects expression, decreases expression	2
bisphenol A	affects expression	1
manganese chloride	increases abundance, increases expression, affects cotreatment	1
perfluorooctane sulfonic acid	increases expression	1
CGP 52608	affects binding, increases reaction	1
K 7174	increases expression	1
abrine	increases expression	1
Air Pollutants	increases abundance, increases expression	1
Azathioprine	increases expression	1
Cadmium	increases expression	1
Calcitriol	increases expression, affects cotreatment	1
Cisplatin	increases expression	1
Doxorubicin	decreases expression	1
Manganese	increases expression, affects cotreatment, increases abundance	1
Methyl Methanesulfonate	increases expression	1
Smoke	decreases expression	1
Dihydrotestosterone	increases expression	1
Testosterone	affects cotreatment, increases expression	1
Copper Sulfate	increases expression	1
Particulate Matter	increases abundance, increases expression	1

Cellosaurus cell lines

2 cell lines: 2 cancer cell line

First 10 cell lines (id-ordered, not curated):

Cellosaurus	Name	Category	Sex
CVCL_TG36	HAP1 PQLC2 (-) 1	Cancer cell line	Male
CVCL_XR81	HAP1 PQLC2 (-) 2	Cancer cell line	Male

Clinical trials (associated diseases)

240 trials via MONDO — disease-level, not drug-specific.

Trial	Phase	Status	Title
NCT00717080	PHASE4	COMPLETED	The Role of Capsular Tension Ring (CTR) in Anterior Capsular Contraction
NCT00000114	PHASE3	COMPLETED	Randomized Trial of Vitamin A and Vitamin E Supplementation for Retinitis Pigmentosa
NCT00000116	PHASE3	COMPLETED	Randomized Trial of DHA for Retinitis Pigmentosa Patients Receiving Vitamin A
NCT00346333	PHASE3	COMPLETED	Clinical Trial of Lutein for Patients With Retinitis Pigmentosa Receiving Vitamin A
NCT01786395	PHASE3	TERMINATED	Phase III Efficacy and Safety Clinical Study of UF-021 for Treatment of Retinitis Pigmentosa
NCT04224207	PHASE3	COMPLETED	Management of Retinitis Pigmentosa by Mesenchymal Stem Cells by Wharton’s Jelly Derived Mesenchymal Stem Cells
NCT04636853	PHASE3	COMPLETED	CB-PRP in Retinitis Pigmentosa and Dry Age-related Macular Degeneration
NCT05537220	PHASE3	ACTIVE_NOT_RECRUITING	Oral N-acetylcysteine for Retinitis Pigmentosa
NCT05800301	PHASE3	COMPLETED	Management of Retinitis Pigmentosa Via Combination of Wharton’s Jelly-derived Mesenchymal Stem Cells and Magnovision
NCT05926583	PHASE3	ACTIVE_NOT_RECRUITING	A Study of AAV5-hRKp.RPGR for the Treatment of Japanese Participants With X-linked Retinitis Pigmentosa
NCT06388200	PHASE3	ACTIVE_NOT_RECRUITING	A Phase 3 Study Of OCU400 Gene Therapy for the Treatment Of Retinitis Pigmentosa
NCT07082855	PHASE3	NOT_YET_RECRUITING	A Multicenter, Randomized, Double-Blind, Controlled Clinical Study of Minocycline for the Treatment of Retinitis Pigmentosa
NCT07290530	PHASE3	NOT_YET_RECRUITING	24-Month Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associated With Usher Syndrome
NCT00100230	PHASE2	COMPLETED	DHA and X-Linked Retinitis Pigmentosa
NCT00447980	PHASE2	COMPLETED	A Study of Encapsulated Cell Technology (ECT) Implant for Participants With Early Stage Retinitis Pigmentosa
NCT00447993	PHASE2	COMPLETED	A Study of Encapsulated Cell Technology (ECT) Implant for Patients With Late Stage Retinitis Pigmentosa
NCT01233609	PHASE2	COMPLETED	Trial of Oral Valproic Acid for Retinitis Pigmentosa
NCT01399515	PHASE2	COMPLETED	Efficacy and Safety of Oral Valproic Acid for Retinitis Pigmentosa
NCT01530659	PHASE2	COMPLETED	Retinal Imaging in CNTF -Releasing Encapsulated Cell Implant Treated Patients for Early-stage Retinitis Pigmentosa
NCT01560715	PHASE2	COMPLETED	Autologous Bone Marrow-Derived Stem Cells Transplantation For Retinitis Pigmentosa
NCT02609165	PHASE2	COMPLETED	Nerve Growth Factor Eye Drops Treatment in Patients With Retinitis Pigmentosa and Cystoid Macular Edema
NCT02661711	PHASE2	COMPLETED	Aflibercept for Macular Oedema With Underlying Retinitis Pigmentosa (AMOUR) Study
NCT02804360	PHASE2	UNKNOWN	Intravitreal Dexamethasone Implant in Retinitis Pigmentosa-related Macular Edema- a Retrospective Study
NCT02837640	PHASE2	UNKNOWN	Studying a Potential Protective Effect of L-Dopa on Retinitis Pigmentosa
NCT03073733	PHASE2	COMPLETED	Safety and Efficacy of Intravitreal Injection of Human Retinal Progenitor Cells in Adults With Retinitis Pigmentosa
NCT04068207	PHASE2	COMPLETED	Minocycline Treatment in Retinitis Pigmentosa
NCT04356716	PHASE2	COMPLETED	Sildenafil for Treatment of Choroidal Ischemia
NCT04604899	PHASE2	COMPLETED	Safety of Repeat Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects With Retinitis Pigmentosa
NCT04763369	PHASE2	UNKNOWN	Investigation of Therapeutic Efficacy and Safety of UMSCs for the Management of Retinitis Pigmentosa (RP)
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