Retinitis Pigmentosa
Watchlist
Retrieved
2022-04-26
Source
Trials
—
Genes
C8orf37,
PDE6B,
PDE6A,
CRX,
RPGR,
RPE65,
PDE6G,
LRAT,
ABCA4,
EYS,
MERTK,
IMPDH1,
ROM1,
RHO,
USH2A,
CRB1,
CNGB1,
RPGRIP1,
GUCY2D,
RP2,
NRL,
RBP3,
CLRN1,
RDH12,
SAG,
SPATA7,
CNGA1,
ARL6,
AIPL1,
REEP6
C8orf37,
PDE6B,
PDE6A,
CRX,
RPGR,
RPE65,
PDE6G,
LRAT,
ABCA4,
EYS,
MERTK,
IMPDH1,
ROM1,
RHO,
USH2A,
CRB1,
CNGB1,
RPGRIP1,
GUCY2D,
RP2,
NRL,
RBP3,
CLRN1,
RDH12,
SAG,
SPATA7,
CNGA1,
ARL6,
AIPL1,
REEP6,
RGR,
DHX38,
GUCA1B,
OFD1,
IDH3A,
IDH3B,
PRPF8,
RP1,
FAM161A,
TULP1,
SNRNP200,
PRPF31,
CERKL,
NR2E3,
CA4,
MAK,
PRCD,
PRPF3,
RLBP1,
PROM1,
PCARE,
CHM,
ARL2BP,
TOPORS,
BBS1,
CYP4V2,
BEST1,
DHDDS,
KLHL7,
IMPG2,
HGSNAT,
IFT140,
PRPF6,
BBS2,
TTC8,
AHI1,
SCAPER,
CLN3,
IFT172,
CDHR1,
KIZ,
FLVCR1,
TTPA,
ARL3,
PRPF4,
AGBL5,
RP9,
SLC7A14,
FSCN2,
POMGNT1,
ZNF513,
ZNF408,
RBP4,
ABHD12,
UNC119,
NEK2,
AHR,
TUB,
SEMA4A,
ATF6,
IFT88,
FOXI2,
UBAP1L,
CCZ1B,
CROCC,
PDAP1,
FAM71A,
KIAA1549,
IRX5,
ARHGEF18,
C1QTNF5,
PRTFDC1,
SLC37A3,
NAALADL1,
CRB2,
NGF,
CEP250,
CWC27,
CCDC66,
GRIN2B,
PRPH2,
AGTPBP1,
SLC6A6,
AIFM1,
FGFR2,
KL,
MT2A,
PTEN,
MYO7A,
CEP290,
GUCA1A,
RDH5,
CDH23,
IQCB1,
MSTO1,
CACNA1A,
BBS4,
ATXN7,
USH1C,
CFAP410,
ATP6,
PANK2,
MKKS,
BBS9,
SLC24A1,
PEX1,
RP1L1,
HADHA,
PNPLA6,
SDCCAG8,
BBS12,
NDUFAF5,
RRM2B,
PDHA1,
NDUFS8,
NDUFV2,
LZTFL1,
FOXRED1,
POMT2,
RCBTB1,
TST,
FKRP,
BBS7,
CNGB3,
NCAPG2,
NPHP1,
MKS1,
NDUFB11,
SURF1,
SDHB,
PEX2,
WDPCP,
PRPH,
NDUFV1,
NDUFA13,
SCN1A,
SCO1,
SDHA,
SDHD,
PHYH,
TACO1,
LIPT1,
SLC19A1,
NDUFA12,
PEX5,
NGLY1,
ALMS1,
LARGE1,
NDUFS4,
PRDX1,
NDUFS3,
POLR3A,
MFSD8,
ZDHHC24,
RNASEH1,
CTNS,
ARL13B,
TRIM32,
NIPAL1,
ECHS1,
FASTKD2,
ERCC3,
POMT1,
ERCC6,
ERG,
IFT27,
NDUFAF6,
BBS5,
NDUFS2,
CLRN1-AS1,
COX20,
CYGB,
COX15,
COX10,
COX8A,
COX7B,
CDH23-AS1,
ACOX1,
C8orf37-AS1,
MMACHC,
JAG1,
AMACR,
AIRE,
PET100,
SDHAF1,
ZFYVE26,
PHF3,
ATP1A2,
BCS1L,
NDUFS7,
CAV1,
TTLL5,
VSX2,
ERCC8,
COX6B1,
PRRT2,
MTFMT,
GSS,
COL18A1,
GMPPB,
HADHB,
ND1,
ND2,
ND3,
ND4,
ND5,
ND6,
TRNK,
TRNL1,
TRNN,
TRNS1,
TRNV,
TRNW,
TRIM37,
BBS10,
NDUFA2,
NDUFA4,
NDUFA9,
NDUFA10,
NDUFS1,
SLC19A3,
WFS1,
BBIP1,
COA8,
HCCS,
NDUFAF2,
HADH,
TMEM14B,
COX14,
PLXNA2,
LSM2,
TRNT1,
CLU,
MFRP,
PCDH15,
DHX16,
PTPRC,
SLU7,
KLK3,
SIGMAR1,
NXNL1,
CLTA,
CNTF,
NT5C2,
RPE,
PROS1,
PLAG1,
NPHP4,
TIMP3,
PSAT1,
NPEPPS,
MYP2,
CXCR6,
RIMS1,
RRH,
SLC19A2,
EXOSC2,
ADIPOR1,
LPAR2,
USP9X,
COG4,
VCP,
EDN1,
LCA5,
PDC,
ATN1,
OTX2,
OTC,
CNOT3,
MVK,
LPCAT1,
MMP9,
EDNRA,
RCC1,
EPO,
INS,
FANCF,
HSPA4,
HK1,
GNAT1,
HIF1A,
OPN4,
GSN,
SERPINF1,
GPR42,
NSMCE3,
GRK1,
ALDH3A2,
SFRP2,
ACKR3,
ADRA1A,
ARL2,
ATXN2,
CC2D2A,
ADRA2B,
WDR19,
SOD3,
PLIN2,
SSTR4,
BRS3,
STC1,
ACTB,
NRG4,
TWIST2,
GRK7,
POC5,
ARMS2,
MFT2,
SAMD11,
SAMD7,
NOC2L,
JAKMIP1,
MIR204,
POLDIP2,
GUCY2EP,
LIN28B,
NPHP3,
ARSI,
RD3,
CENPV,
PITPNM3,
INVS,
CENPK,
TENT5A,
CYCS,
DCUN1D1,
TWNK,
SLC2A4RG,
TBX20,
RDH11,
PNPLA2,
KIDINS220,
NGB,
MPP4,
NYX,
TNMD,
ENFL2,
TUT1,
DNER,
FTO,
ELOVL6,
ALG12,
RNF19A,
COQ8B,
SETD2,
KLF15,
PDZD7,
HKDC1,
C5AR2,
DNAJC17,
ADGRV1,
CEP78,
GNPTG,
AAVS1,
THBS2,
WHRN,
MMP2,
HMOX1,
HK2,
HGF,
GRM5,
GRM1,
GRB10,
GLO1,
GK,
GJB2,
GDNF,
GDF2,
G6PD,
FRZB,
FN1,
FGF5,
FGF2,
FBN2,
HSP90AA1,
IDH2,
IFNG,
INSR,
MEIS2,
MDH1,
MAP1A,
SMAD4,
LTB,
LAMP1,
ISG20,
ING1,
IGF1,
IMPA1,
CXCL8,
IL6,
IL2RA,
IL1B,
IL1A,
CCN1,
FASN,
ETV5,
ERN1,
ALDH7A1,
CACNA1F,
C5AR1,
C3,
C1QBP,
BSG,
BMP4,
BCL2,
ARRB2,
CANX,
ARR3,
ABCC6,
APOE,
APOB,
AMFR,
ADH7,
ABO,
CACNA1S,
CCT,
ERBB2,
CYBB,
EGF,
E2F1,
DUSP6,
DNMT3A,
CFD,
ACE,
CYLD,
CTNNB1,
CD44,
MAPK14,
CRYAB,
CRK,
CP,
CORD1,
COL2A1,
CD74,
RAB8A,
MSR1,
SH3BP4,
CYTB,
SYNJ1,
FGF18,
HSD17B6,
DGKE,
BRAP,
SMC1A,
USP11,
AIMP2,
PAX8,
ZNF132,
ZFP36,
USH1E,
TSC1,
TP53,
TNF,
TMPRSS2,
TSPAN7,
SMC3,
ARHGEF2,
CRLF1,
AKT3,
TMED3,
SIRT1,
ARC,
MAPRE3,
ARPP21,
AHSA1,
PPIH,
HEPH,
SNAP29,
PLEKHM1,
PHYHIP,
HDAC4,
KNTC1,
EIF2AK3,
GRAP2,
EFTUD2,
TIMP2,
TIMP1,
DYNLT3,
PKNOX1,
HTRA1,
PRNP,
MAPK1,
PRKCG,
POLG,
PMM2,
PLAU,
PIM1,
RAC1,
PGF,
CFP,
PDGFRB,
NPTX2,
NFE2L2,
NAGLU,
MYC,
ALDH18A1,
RASGRF1,
ELOVL4,
SFTPD,
STATH,
SOS1,
SOD1,
SNRPB,
SNCA,
SLC2A1,
SGSH,
SFRP5,
RBP1,
SEC14L1,
CX3CL1,
CCL2,
S100A6,
RPS6KB1,
RPS6,
RCVRN,
H3P22
Drugs
17-(Dimethylaminoethylamino)-17-demethoxygeldanamycin (after administration of adeno-associated viral vector encoding an inducible short hairpin RNA targeting claudin-5),
2'-O-(2-Methoxyethyl)-modified antisense oligonucleotide targeting exon 13 in the USH2A gene,
3-[(4aR,6R,7R,7aS)-7-hydroxy-2-oxido-2-sulfanylidene-4a,6,7,7a-tetrahydro-4H-furo [3,2-d][1,3,2] dioxaphosphinin-6-yl]-2-bromo-6-phenyl-5H-imidazo[1,2-a]purin-9-one
17-(Dimethylaminoethylamino)-17-demethoxygeldanamycin (after administration of adeno-associated viral vector encoding an inducible short hairpin RNA targeting claudin-5),
2'-O-(2-Methoxyethyl)-modified antisense oligonucleotide targeting exon 13 in the USH2A gene,
3-[(4aR,6R,7R,7aS)-7-hydroxy-2-oxido-2-sulfanylidene-4a,6,7,7a-tetrahydro-4H-furo [3,2-d][1,3,2] dioxaphosphinin-6-yl]-2-bromo-6-phenyl-5H-imidazo[1,2-a]purin-9-one,
4,7,10,13,16,19-Docosahexaenoic acid,
4-[(2E)-1-oxo-3-(2,6,6-trimethyl-1-cyclohexen-1-yl)-2-propen-1-yl]-1-piperazinecarboxamide,
9-cis-Retinyl acetate,
Adeno-associated viral vector containing DNA encoding an RNAi targeting rhodopsin / adeno-associated viral vector containing a rhodopsin gene,
Adeno-associated viral vector encoding an inducible short hairpin RNA targeting claudin-5 (prior to administration of 17-dimethylaminoethylamino-17-demethocygeldanamycin),
Adeno-associated viral vector serotype 2.7m8 containing the ChrimsonR-tdTomato gene,
Adeno-associated viral vector serotype 8 encoding engineered rhodopsin DNA-binding repressor and human rhodopsin expression cassettes,
Adeno-associated virus serotype 8 containing the human RdCVF sequence and the human RdCVFL sequence,
Adenovirus associated viral vector serotype 4 containing the human RPE65 gene,
Adenovirus associated viral vector serotype 5 containing the human pde6 beta gene,
Adenovirus-associated viral vector serotype 2 containing the human RPE65 gene (AAV2-hRPE65v2 ),
Adenovirus-associated viral vector serotype 8 containing the human RPGR gene,
All-cis-docosa-4,7,10,13,16,19-hexaenoic acid,
Allogeneic fetal human retinal progenitor cells expanded ex vivo,
Allogeneic human umbilical cord tissue-derived cells,
Allogeneic retinal epithelial cells transfected with plasmid vector expressing ciliary neurotrophic growth,
Antisense oligonucleotide targeting exon 13 in the USH2A gene,
Antisense oligonucleotide targeting the USH2A gene,
Combination of three adeno-associated viral vectors of serotype 8 containing the 5'-, the body- and the 3'- coding sequences of human CEP290 fused to inteins,
Encapsulated human retinal pigment epithelial cell line transfected with plasmid vector expressing human ciliary neurotropic factor,
Expanded human allogeneic neural retinal progenitor cells extracted from neural retina,
Human Umbilical Tissue-Derived Cells,
Myriocin,
Non-replicating recombinant adeno-associated virus vector containing a fragment of the gene encoding channelrhodopsin-2 protein,
Recombinant adeno-associated viral vector containing the human RPGR gene,
Recombinant human mesencephalic astrocyte-derived neurotrophic factor (MANF),
Recombinant human methionine proinsulin,
Recombinant human nerve growth factor
(
OXERVATE
),
Recombinant human proinsulin,
Recombinant human rod-derived cone viability factor,
Recombinant lens epithelium derived growth factor 1-326 (LEDGF1-326),
Unoprostone isopropyl,
adenovirus associated viral vector serotype 5 containing the RPGR gene
Registered!

Retinitis pigmentosa (RP) is a group of inherited eye diseases that affect the light-sensitive part of the eye (retina). RP causes cells in the retina to die, causing progressive vision loss. The first sign of RP usually is night blindness. As the condition progresses, affected individuals develop tunnel vision (loss of peripheral vision), and eventually loss of central vision. RP may be caused by mutations in any of at least 50 genes. Inheritance can be autosomal dominant, autosomal recessive, or X-linked. Treatment options to slow the progression of vision loss include light avoidance, use of low-vision aids, and vitamin A supplementation. Researchers are working to develop new treatment options for the future such as gene therapy, stem cell transplantation and prosthetic implants.