Personalized therapeutic strategies for patients with retinitis pigmentosa

Retinitis pigmentosa (RP) encompasses many different hereditary retinal degenerations that are caused by a vast array of different gene mutations and have highly variable disease presentations and severities. This heterogeneity poses a significant therapeutic challenge, although an answer may eventu...

Full description

Saved in:
Bibliographic Details
Published inExpert opinion on biological therapy Vol. 15; no. 3; p. 391
Main Authors Zheng, Andrew, Li, Yao, Tsang, Stephen H
Format Journal Article
LanguageEnglish
Published England 01.03.2015
Subjects
Clustered Regularly Interspaced Short Palindromic Repeats - genetics
Genetic Therapy - methods
Genetic Therapy - trends
Humans
Induced Pluripotent Stem Cells - transplantation
Precision Medicine - methods
Precision Medicine - trends
Retinal Pigment Epithelium - pathology
Retinitis Pigmentosa - diagnosis
Retinitis Pigmentosa - genetics
Retinitis Pigmentosa - therapy
Stem Cell Transplantation - methods
Stem Cell Transplantation - trends
clustered regularly interspaced short palindromic repeats
gene therapy
induced pluripotent stem cells
retinal degenerations
Online AccessGet more information

Cover

Abstract Retinitis pigmentosa (RP) encompasses many different hereditary retinal degenerations that are caused by a vast array of different gene mutations and have highly variable disease presentations and severities. This heterogeneity poses a significant therapeutic challenge, although an answer may eventually be found through two recent innovations: induced pluripotent stem cells (iPSCs) and clustered regularly interspaced short palindromic repeats (CRISPR)/Cas genome editing. This review discusses the wide-ranging applications of iPSCs and CRISPR-including disease modelling, diagnostics and therapeutics - with an ultimate view towards understanding how these two technologies can come together to address disease heterogeneity and orphan genes in a novel personalized medicine platform. An extensive literature search was conducted in PubMed and Google Scholar, with a particular focus on high-impact research published within the last 1 - 2 years and centered broadly on the subjects of retinal gene therapy, iPSC-derived outer retina cells, stem cell transplantation and CRISPR/Cas gene editing. For the retinal pigment epithelium, autologous transplantation of gene-corrected grafts derived from iPSCs may well be technically feasible in the near future. Photoreceptor transplantation faces more significant unresolved technical challenges but remains an achievable, if more distant, goal given the rapid pace of advancements in the field.
AbstractList Retinitis pigmentosa (RP) encompasses many different hereditary retinal degenerations that are caused by a vast array of different gene mutations and have highly variable disease presentations and severities. This heterogeneity poses a significant therapeutic challenge, although an answer may eventually be found through two recent innovations: induced pluripotent stem cells (iPSCs) and clustered regularly interspaced short palindromic repeats (CRISPR)/Cas genome editing. This review discusses the wide-ranging applications of iPSCs and CRISPR-including disease modelling, diagnostics and therapeutics - with an ultimate view towards understanding how these two technologies can come together to address disease heterogeneity and orphan genes in a novel personalized medicine platform. An extensive literature search was conducted in PubMed and Google Scholar, with a particular focus on high-impact research published within the last 1 - 2 years and centered broadly on the subjects of retinal gene therapy, iPSC-derived outer retina cells, stem cell transplantation and CRISPR/Cas gene editing. For the retinal pigment epithelium, autologous transplantation of gene-corrected grafts derived from iPSCs may well be technically feasible in the near future. Photoreceptor transplantation faces more significant unresolved technical challenges but remains an achievable, if more distant, goal given the rapid pace of advancements in the field.
Author Li, Yao
Zheng, Andrew
Tsang, Stephen H
Author_xml – sequence: 1
  givenname: Andrew
  surname: Zheng
  fullname: Zheng, Andrew
  organization: Columbia University, College of Physicians and Surgeons , 50 Haven Ave, Box #123, Bard Hall, New York, NY 10032 , USA
– sequence: 2
  givenname: Yao
  surname: Li
  fullname: Li, Yao
– sequence: 3
  givenname: Stephen H
  surname: Tsang
  fullname: Tsang, Stephen H
BackLink https://www.ncbi.nlm.nih.gov/pubmed/25613576$$D View this record in MEDLINE/PubMed
BookMark eNo1j8lKBDEURYMo9qCfoOQHqsx7maqW0jjSoAtdN6nKq-5I10CSRvTrbVBXF87icO6CnQ7jQIxdgShBg70BZQF1XZUoQJcghIEaT9gcrFKFNRXO2CKlDyFQ1BrP2Qy1AamtmbPnV4ppHNw-fJPneUfRTXTIoeUpR5dpGyjxbox8cjnQkBP_DHnHI-UwhBwSn8K2P_IxuQt21rl9osu_XbL3-7u31WOxfnl4Wt2ui1ajyoWRKFELEpUGVxlQrlVgj2miaRvwSkol20p22osOsBPoJCnV-MY509na45Jd_3qnQ9OT30wx9C5-bf5P4Q9amFBT
CitedBy_id crossref_primary_10_1080_13816810_2024_2423784
crossref_primary_10_1155_2017_1610691
crossref_primary_10_1016_j_scr_2019_101468
crossref_primary_10_1016_j_scr_2019_101385
crossref_primary_10_1126_scitranslmed_aaf2838
crossref_primary_10_1038_mtm_2016_10
crossref_primary_10_1007_s00439_018_1933_9
crossref_primary_10_1038_srep41086
crossref_primary_10_1152_physiolgenomics_00096_2016
crossref_primary_10_1038_eye_2015_115
crossref_primary_10_1080_01677063_2017_1395876
crossref_primary_10_1038_mp_2016_89
crossref_primary_10_1097_APO_0000000000000227
crossref_primary_10_23950_jcmk_11931
crossref_primary_10_3390_pharmaceutics13060865
crossref_primary_10_2217_rme_2016_0156
crossref_primary_10_1007_s10787_024_01506_y
crossref_primary_10_1186_s12967_017_1183_y
crossref_primary_10_3928_23258160_20151214_01
crossref_primary_10_1155_2019_5743109
crossref_primary_10_1146_annurev_vision_100419_111701
crossref_primary_10_1016_j_exer_2022_108961
crossref_primary_10_1016_j_heliyon_2023_e22443
crossref_primary_10_1002_mgg3_266
crossref_primary_10_1080_02713683_2017_1358371
crossref_primary_10_1080_23262133_2017_1324258
crossref_primary_10_1016_j_preteyeres_2017_10_004
crossref_primary_10_1016_j_omtm_2019_11_016
crossref_primary_10_7759_cureus_67046
crossref_primary_10_3389_fped_2018_00023
crossref_primary_10_3389_fmed_2024_1468230
crossref_primary_10_1007_s12015_024_10689_4
crossref_primary_10_1016_j_oret_2017_11_010
crossref_primary_10_1155_2016_2850873
ContentType Journal Article
DBID CGR
CUY
CVF
ECM
EIF
NPM
DOI 10.1517/14712598.2015.1006192
DatabaseName Medline
MEDLINE
MEDLINE (Ovid)
MEDLINE
MEDLINE
PubMed
DatabaseTitle MEDLINE
Medline Complete
MEDLINE with Full Text
PubMed
MEDLINE (Ovid)
DatabaseTitleList MEDLINE
Database_xml – sequence: 1
  dbid: NPM
  name: PubMed
  url: https://proxy.k.utb.cz/login?url=http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?db=PubMed
  sourceTypes: Index Database
– sequence: 2
  dbid: EIF
  name: MEDLINE
  url: https://proxy.k.utb.cz/login?url=https://www.webofscience.com/wos/medline/basic-search
  sourceTypes: Index Database
DeliveryMethod no_fulltext_linktorsrc
Discipline Pharmacy, Therapeutics, & Pharmacology
EISSN 1744-7682
ExternalDocumentID 25613576
Genre Review
Research Support, Non-U.S. Gov't
Journal Article
Research Support, N.I.H., Extramural
GrantInformation_xml – fundername: NCI NIH HHS
  grantid: 5P30CA013696
– fundername: NEI NIH HHS
  grantid: R01 EY018213
– fundername: NCI NIH HHS
  grantid: P30 CA013696
– fundername: NEI NIH HHS
  grantid: R01EY018213
– fundername: NEI NIH HHS
  grantid: 5P30EY019007
– fundername: NEI NIH HHS
  grantid: P30 EY019007
GroupedDBID ---
00X
03L
0R~
0VX
29G
4.4
53G
5GY
AAMIU
AAOUU
AAPWH
ABBAB
ABEIZ
ABJNI
ABJYH
ABLIJ
ABLKL
ABXYU
ACGFS
ACIEZ
ADCVX
ADRBQ
ADTOD
AECIN
AENEX
AEOZL
AFRVT
AGDLA
AGMHR
AGMLL
AIJEM
AIRBT
AIYSM
AIZAD
AKBVH
ALMA_UNASSIGNED_HOLDINGS
ALQZU
BABNJ
BLEHA
CAZVN
CCCUG
CGR
CS3
CUY
CVF
DASJU
DAWQK
DKSSO
DU5
EBS
ECM
EIF
EJD
EMOBN
F5P
H13
HZ~
KRBQP
KSSTO
KWAYT
KYCEM
LJTGL
M44
M4Z
NPM
O9-
P2P
RNANH
TASJS
TBQAZ
TDBHL
TERGH
TFDNU
TFL
TFW
TUROJ
TZHSB
V1S
~1N
ID FETCH-LOGICAL-c524t-6323250e0851a8614ac4170200bcb1d43343c83f5d0f12f02a3e44bdbaa6f79d2
IngestDate Mon Jul 21 06:02:12 EDT 2025
IsDoiOpenAccess false
IsOpenAccess true
IsPeerReviewed true
IsScholarly true
Issue 3
Keywords clustered regularly interspaced short palindromic repeats
gene therapy
induced pluripotent stem cells
retinal degenerations
Language English
LinkModel OpenURL
MergedId FETCHMERGED-LOGICAL-c524t-6323250e0851a8614ac4170200bcb1d43343c83f5d0f12f02a3e44bdbaa6f79d2
OpenAccessLink http://doi.org/10.1517/14712598.2015.1006192
PMID 25613576
ParticipantIDs pubmed_primary_25613576
PublicationCentury 2000
PublicationDate 2015-03-01
PublicationDateYYYYMMDD 2015-03-01
PublicationDate_xml – month: 03
  year: 2015
  text: 2015-03-01
  day: 01
PublicationDecade 2010
PublicationPlace England
PublicationPlace_xml – name: England
PublicationTitle Expert opinion on biological therapy
PublicationTitleAlternate Expert Opin Biol Ther
PublicationYear 2015
References 21475194 - Nature. 2011 Apr 7;472(7341):51-6
25002228 - Neuron. 2014 Aug 6;83(3):586-600
23248312 - Proc Natl Acad Sci U S A. 2013 Jan 2;110(1):354-9
25203033 - Nat Biotechnol. 2014 Sep;32(9):874-6
23315928 - Curr Protoc Hum Genet. 2013 Jan;Chapter 7:Unit7.20
21347327 - PLoS One. 2011;6(2):e17084
18774912 - Hum Gene Ther. 2008 Oct;19(10):979-90
17093405 - Nature. 2006 Nov 9;444(7116):203-7
8942864 - Ophthalmology. 1996 Nov;103(11):1732-43
23900607 - Invest Ophthalmol Vis Sci. 2013;54(9):6118-32
23808604 - Hum Gene Ther Clin Dev. 2013 Jun;24(2):55-64
23845948 - Science. 2013 Aug 23;341(6148):1233158
23974631 - Sci Rep. 2013;3:2510
22281388 - Lancet. 2012 Feb 25;379(9817):713-20
25049410 - Proc Natl Acad Sci U S A. 2014 Aug 5;111(31):11461-6
18441370 - N Engl J Med. 2008 May 22;358(21):2240-8
19854499 - Lancet. 2009 Nov 7;374(9701):1597-605
23991284 - Elife. 2013;2:e00824
9409224 - Arterioscler Thromb Vasc Biol. 1997 Nov;17(11):2532-9
16904174 - Cell. 2006 Aug 25;126(4):663-76
21966470 - PLoS One. 2011;6(9):e25255
24646489 - Stem Cells Transl Med. 2014 May;3(5):586-98
23287718 - Science. 2013 Feb 15;339(6121):819-23
20535210 - Nature. 2010 Jun 10;465(7299):808-12
23467559 - Sci Transl Med. 2013 Mar 6;5(175):175fs8
24439297 - Lancet. 2014 Mar 29;383(9923):1129-37
20881025 - Br J Ophthalmol. 2011 Apr;95(4):539-43
23701314 - Clin Genet. 2013 Aug;84(2):132-41
11393491 - MMWR Morb Mortal Wkly Rep. 2001 Feb 23;50(7):120-5
6534229 - An Acad Bras Cienc. 1984 Dec;56(4):431-43
24807808 - Gene Ther. 2014 Jul;21(7):662-72
21706032 - Nature. 2011 Jul 14;475(7355):217-21
23907171 - Nat Biotechnol. 2013 Sep;31(9):833-8
25458728 - Lancet. 2015 Feb 7;385(9967):509-16
24534198 - Exp Eye Res. 2014 Jun;123:161-72
11326284 - Nat Genet. 2001 May;28(1):92-5
23288902 - Proc Natl Acad Sci U S A. 2013 Jan 15;110(3):1101-6
22570183 - Stem Cells. 2012 Jul;30(7):1424-35
25418680 - Mol Ther. 2015 Mar;23(3):570-7
1418997 - Neuron. 1992 Nov;9(5):815-30
24936453 - Stem Cell Reports. 2014 May 6;2(5):662-74
12414111 - Brain Res. 2002 Nov 8;954(2):286-93
25186301 - Biotechnol J. 2014 Nov;9(11):1402-12
9786999 - J Neurosci. 1998 Nov 1;18(21):8936-46
25448922 - Prog Retin Eye Res. 2015 Jan;44:15-35
15806097 - Nature. 2005 Jun 2;435(7042):646-51
19128794 - Cell Stem Cell. 2009 Jan 9;4(1):73-9
23992846 - Cell. 2013 Sep 12;154(6):1380-9
23000899 - Nature. 2012 Nov 1;491(7422):114-8
22745249 - Science. 2012 Aug 17;337(6096):816-21
23643243 - Cell. 2013 May 9;153(4):910-8
24463574 - Nat Biotechnol. 2014 Mar;32(3):279-84
24101599 - Hum Mol Genet. 2014 Jan 15;23(2):514-23
8512476 - Arch Ophthalmol. 1993 Jun;111(6):761-72
18522845 - Cell Stem Cell. 2008 Jun 5;2(6):525-8
18441371 - N Engl J Med. 2008 May 22;358(21):2231-9
23873086 - Nat Biotechnol. 2013 Aug;31(8):741-7
21491544 - Stem Cells. 2011 Jun;29(6):972-80
15781279 - Exp Eye Res. 2005 Apr;80(4):515-25
23918387 - Proc Natl Acad Sci U S A. 2013 Aug 20;110(34):13904-9
20005794 - Cell. 2009 Dec 11;139(6):1032-7
18035408 - Cell. 2007 Nov 30;131(5):861-72
18059259 - Nat Biotechnol. 2008 Jan;26(1):101-6
23667438 - PLoS One. 2013;8(5):e61396
22522934 - Nature. 2012 May 3;485(7396):99-103
25292197 - Hum Mol Genet. 2015 Feb 15;24(4):972-86
24895994 - Mol Ther. 2014 Sep;22(9):1688-97
9085110 - Graefes Arch Clin Exp Ophthalmol. 1997 Mar;235(3):149-58
20671281 - Invest Ophthalmol Vis Sci. 2010 Dec;51(12):6671-80
22539871 - Mol Vis. 2012;18:920-36
25205868 - Invest Ophthalmol Vis Sci. 2014 Oct;55(10):6301-8
21179091 - Nat Biotechnol. 2011 Feb;29(2):143-8
20098701 - PLoS One. 2010;5(1):e8763
24935155 - Mol Brain. 2014;7:45
23341635 - Proc Natl Acad Sci U S A. 2013 Feb 5;110(6):E517-25
20648062 - Nat Rev Neurosci. 2010 Aug;11(8):563-76
19358155 - Genesis. 2009 May;47(5):314-22
12657607 - Invest Ophthalmol Vis Sci. 2003 Apr;44(4):1663-72
20385935 - Arch Ophthalmol. 2010 Apr;128(4):403-11
22895806 - Mol Med. 2012;18:1312-9
23561441 - Cell Stem Cell. 2013 Apr 4;12(4):393-4
21900357 - Hum Mol Genet. 2011 Dec 1;20(23):4530-9
19481515 - Cell Stem Cell. 2009 Jun 5;4(6):472-6
21240260 - Nature. 2011 Mar 10;471(7337):225-9
21825139 - Proc Natl Acad Sci U S A. 2011 Aug 23;108(34):E569-76
23845947 - Science. 2013 Aug 23;341(6148):1233151
15505071 - Invest Ophthalmol Vis Sci. 2004 Nov;45(11):4167-73
20378608 - Hum Mol Genet. 2010 Jul 1;19(13):2581-93
21235327 - Hum Gene Ther. 2011 May;22(5):537-47
23544203 - JAMA Ophthalmol. 2013 Feb;131(2):183-9
17554338 - Nature. 2007 Jul 19;448(7151):313-7
21559507 - PLoS One. 2011;6(4):e18992
10995547 - Mol Cell Neurosci. 2000 Sep;16(3):197-205
19060896 - Nat Neurosci. 2009 Jan;12(1):44-52
24647603 - Hum Mol Genet. 2014 Sep 15;23(R1):R9-R16
22949671 - Proc Natl Acad Sci U S A. 2012 Sep 25;109(39):E2579-86
References_xml – reference: 24935155 - Mol Brain. 2014;7:45
– reference: 18774912 - Hum Gene Ther. 2008 Oct;19(10):979-90
– reference: 24534198 - Exp Eye Res. 2014 Jun;123:161-72
– reference: 20385935 - Arch Ophthalmol. 2010 Apr;128(4):403-11
– reference: 23341635 - Proc Natl Acad Sci U S A. 2013 Feb 5;110(6):E517-25
– reference: 22539871 - Mol Vis. 2012;18:920-36
– reference: 25002228 - Neuron. 2014 Aug 6;83(3):586-600
– reference: 23974631 - Sci Rep. 2013;3:2510
– reference: 19358155 - Genesis. 2009 May;47(5):314-22
– reference: 8512476 - Arch Ophthalmol. 1993 Jun;111(6):761-72
– reference: 19481515 - Cell Stem Cell. 2009 Jun 5;4(6):472-6
– reference: 25458728 - Lancet. 2015 Feb 7;385(9967):509-16
– reference: 6534229 - An Acad Bras Cienc. 1984 Dec;56(4):431-43
– reference: 23287718 - Science. 2013 Feb 15;339(6121):819-23
– reference: 25203033 - Nat Biotechnol. 2014 Sep;32(9):874-6
– reference: 1418997 - Neuron. 1992 Nov;9(5):815-30
– reference: 17554338 - Nature. 2007 Jul 19;448(7151):313-7
– reference: 22895806 - Mol Med. 2012;18:1312-9
– reference: 20535210 - Nature. 2010 Jun 10;465(7299):808-12
– reference: 23000899 - Nature. 2012 Nov 1;491(7422):114-8
– reference: 24101599 - Hum Mol Genet. 2014 Jan 15;23(2):514-23
– reference: 24647603 - Hum Mol Genet. 2014 Sep 15;23(R1):R9-R16
– reference: 21179091 - Nat Biotechnol. 2011 Feb;29(2):143-8
– reference: 22570183 - Stem Cells. 2012 Jul;30(7):1424-35
– reference: 23992846 - Cell. 2013 Sep 12;154(6):1380-9
– reference: 11393491 - MMWR Morb Mortal Wkly Rep. 2001 Feb 23;50(7):120-5
– reference: 16904174 - Cell. 2006 Aug 25;126(4):663-76
– reference: 24463574 - Nat Biotechnol. 2014 Mar;32(3):279-84
– reference: 23701314 - Clin Genet. 2013 Aug;84(2):132-41
– reference: 22949671 - Proc Natl Acad Sci U S A. 2012 Sep 25;109(39):E2579-86
– reference: 24807808 - Gene Ther. 2014 Jul;21(7):662-72
– reference: 24936453 - Stem Cell Reports. 2014 May 6;2(5):662-74
– reference: 23561441 - Cell Stem Cell. 2013 Apr 4;12(4):393-4
– reference: 18522845 - Cell Stem Cell. 2008 Jun 5;2(6):525-8
– reference: 25205868 - Invest Ophthalmol Vis Sci. 2014 Oct;55(10):6301-8
– reference: 8942864 - Ophthalmology. 1996 Nov;103(11):1732-43
– reference: 24439297 - Lancet. 2014 Mar 29;383(9923):1129-37
– reference: 21240260 - Nature. 2011 Mar 10;471(7337):225-9
– reference: 23873086 - Nat Biotechnol. 2013 Aug;31(8):741-7
– reference: 25049410 - Proc Natl Acad Sci U S A. 2014 Aug 5;111(31):11461-6
– reference: 21347327 - PLoS One. 2011;6(2):e17084
– reference: 21235327 - Hum Gene Ther. 2011 May;22(5):537-47
– reference: 24646489 - Stem Cells Transl Med. 2014 May;3(5):586-98
– reference: 23900607 - Invest Ophthalmol Vis Sci. 2013;54(9):6118-32
– reference: 9409224 - Arterioscler Thromb Vasc Biol. 1997 Nov;17(11):2532-9
– reference: 23808604 - Hum Gene Ther Clin Dev. 2013 Jun;24(2):55-64
– reference: 21966470 - PLoS One. 2011;6(9):e25255
– reference: 19128794 - Cell Stem Cell. 2009 Jan 9;4(1):73-9
– reference: 21825139 - Proc Natl Acad Sci U S A. 2011 Aug 23;108(34):E569-76
– reference: 23845948 - Science. 2013 Aug 23;341(6148):1233158
– reference: 21559507 - PLoS One. 2011;6(4):e18992
– reference: 23315928 - Curr Protoc Hum Genet. 2013 Jan;Chapter 7:Unit7.20
– reference: 21900357 - Hum Mol Genet. 2011 Dec 1;20(23):4530-9
– reference: 9085110 - Graefes Arch Clin Exp Ophthalmol. 1997 Mar;235(3):149-58
– reference: 19060896 - Nat Neurosci. 2009 Jan;12(1):44-52
– reference: 25448922 - Prog Retin Eye Res. 2015 Jan;44:15-35
– reference: 20378608 - Hum Mol Genet. 2010 Jul 1;19(13):2581-93
– reference: 17093405 - Nature. 2006 Nov 9;444(7116):203-7
– reference: 23845947 - Science. 2013 Aug 23;341(6148):1233151
– reference: 23288902 - Proc Natl Acad Sci U S A. 2013 Jan 15;110(3):1101-6
– reference: 18441370 - N Engl J Med. 2008 May 22;358(21):2240-8
– reference: 23544203 - JAMA Ophthalmol. 2013 Feb;131(2):183-9
– reference: 22522934 - Nature. 2012 May 3;485(7396):99-103
– reference: 15806097 - Nature. 2005 Jun 2;435(7042):646-51
– reference: 25186301 - Biotechnol J. 2014 Nov;9(11):1402-12
– reference: 18059259 - Nat Biotechnol. 2008 Jan;26(1):101-6
– reference: 23907171 - Nat Biotechnol. 2013 Sep;31(9):833-8
– reference: 10995547 - Mol Cell Neurosci. 2000 Sep;16(3):197-205
– reference: 20005794 - Cell. 2009 Dec 11;139(6):1032-7
– reference: 23667438 - PLoS One. 2013;8(5):e61396
– reference: 20671281 - Invest Ophthalmol Vis Sci. 2010 Dec;51(12):6671-80
– reference: 20648062 - Nat Rev Neurosci. 2010 Aug;11(8):563-76
– reference: 22281388 - Lancet. 2012 Feb 25;379(9817):713-20
– reference: 20098701 - PLoS One. 2010;5(1):e8763
– reference: 18441371 - N Engl J Med. 2008 May 22;358(21):2231-9
– reference: 22745249 - Science. 2012 Aug 17;337(6096):816-21
– reference: 12657607 - Invest Ophthalmol Vis Sci. 2003 Apr;44(4):1663-72
– reference: 9786999 - J Neurosci. 1998 Nov 1;18(21):8936-46
– reference: 21706032 - Nature. 2011 Jul 14;475(7355):217-21
– reference: 24895994 - Mol Ther. 2014 Sep;22(9):1688-97
– reference: 23918387 - Proc Natl Acad Sci U S A. 2013 Aug 20;110(34):13904-9
– reference: 15781279 - Exp Eye Res. 2005 Apr;80(4):515-25
– reference: 23467559 - Sci Transl Med. 2013 Mar 6;5(175):175fs8
– reference: 23643243 - Cell. 2013 May 9;153(4):910-8
– reference: 15505071 - Invest Ophthalmol Vis Sci. 2004 Nov;45(11):4167-73
– reference: 23991284 - Elife. 2013;2:e00824
– reference: 23248312 - Proc Natl Acad Sci U S A. 2013 Jan 2;110(1):354-9
– reference: 21491544 - Stem Cells. 2011 Jun;29(6):972-80
– reference: 12414111 - Brain Res. 2002 Nov 8;954(2):286-93
– reference: 25418680 - Mol Ther. 2015 Mar;23(3):570-7
– reference: 18035408 - Cell. 2007 Nov 30;131(5):861-72
– reference: 20881025 - Br J Ophthalmol. 2011 Apr;95(4):539-43
– reference: 25292197 - Hum Mol Genet. 2015 Feb 15;24(4):972-86
– reference: 19854499 - Lancet. 2009 Nov 7;374(9701):1597-605
– reference: 11326284 - Nat Genet. 2001 May;28(1):92-5
– reference: 21475194 - Nature. 2011 Apr 7;472(7341):51-6
SSID ssj0020952
Score 2.2782562
SecondaryResourceType review_article
Snippet Retinitis pigmentosa (RP) encompasses many different hereditary retinal degenerations that are caused by a vast array of different gene mutations and have...
SourceID pubmed
SourceType Index Database
StartPage 391
SubjectTerms Clustered Regularly Interspaced Short Palindromic Repeats - genetics
Genetic Therapy - methods
Genetic Therapy - trends
Humans
Induced Pluripotent Stem Cells - transplantation
Precision Medicine - methods
Precision Medicine - trends
Retinal Pigment Epithelium - pathology
Retinitis Pigmentosa - diagnosis
Retinitis Pigmentosa - genetics
Retinitis Pigmentosa - therapy
Stem Cell Transplantation - methods
Stem Cell Transplantation - trends
Title Personalized therapeutic strategies for patients with retinitis pigmentosa
URI https://www.ncbi.nlm.nih.gov/pubmed/25613576
Volume 15
hasFullText
inHoldings 1
isFullTextHit
isPrint
link http://utb.summon.serialssolutions.com/2.0.0/link/0/eLvHCXMwnV1NT8JAEN2IXrwYv7_NHgwXKLbd3bYcjdEQEg0HSNAL2W636kEgaS_w6539KC2IRr00pAtN23lM3kzevEXoOqJR0I487nDOiUNDQpwoSnyHS58K4nIq9JYsj09BZ0C7QzYsBZl6uiSPW2K-dq7kP1GFcxBXNSX7h8guLgon4DPEF44QYTj-Ksa9gknPpZZBFpNUjSwvHCC0jNCap9pJNjW3qCRDWWP6_qqag5OMLzXolftx3lCTVJpNjhvGqUmHM192IXh5kyZZVISRSt6jNQLPfLJoDGRFX9qoyuxMhG03eKzUW7WkSZEhpQ4UKcs5lFWwQioJkZi9uL4kauaZUf8QCFZbS-yYEmyocq76fXjf0w8dPV9VOszsFfPz6op_drFUQzWoJNTWqKqfY0tyIJi-neuCO7pZez_KL9peY6X20Bykv4t2bPGAbw0S9tCGHO-jes-4j8-auF9CIGviOu6VvuSzA9StwgVX4IJLuGCACy7gghVc8AIuuITLIRo83PfvOo7dSsMRzKe5ExBgzsyVimDzCCgZF9QL4fndWMReQgmhREQkZYmben7q-pxISuMk5jxIw3biH6HN8WQsTxBOgIIzoNWpgN8pU1kWC6j5eRokkN5deYqOzTsaTY1fyqh4e2ffrpyj7RJrF2grhT-ovAS2l8dXOmCf7SlUiA
linkProvider National Library of Medicine
openUrl ctx_ver=Z39.88-2004&ctx_enc=info%3Aofi%2Fenc%3AUTF-8&rfr_id=info%3Asid%2Fsummon.serialssolutions.com&rft_val_fmt=info%3Aofi%2Ffmt%3Akev%3Amtx%3Ajournal&rft.genre=article&rft.atitle=Personalized+therapeutic+strategies+for+patients+with+retinitis+pigmentosa&rft.jtitle=Expert+opinion+on+biological+therapy&rft.au=Zheng%2C+Andrew&rft.au=Li%2C+Yao&rft.au=Tsang%2C+Stephen+H&rft.date=2015-03-01&rft.eissn=1744-7682&rft.volume=15&rft.issue=3&rft.spage=391&rft_id=info:doi/10.1517%2F14712598.2015.1006192&rft_id=info%3Apmid%2F25613576&rft_id=info%3Apmid%2F25613576&rft.externalDocID=25613576