Læknablaðið - 01.12.2015, Blaðsíða 30
586 LÆKNAblaðið 2015/101
ENgLISH SUMMArY
Embryonic stem cells are, as the name indicates, isolated from embryos.
They are pluripotent cells which can be maintained undifferentiated
or induced to differentiate into any cell type of the body. In 1998 the
first isolation of human embryonic stem cells was successful and they
became an interesting source for stem cell regenerative medicine. only
8 years later pluripotent stem cells were generated by reprogramming
somatic cells into induced pluripotent stem cells (iPSCs). This was a
revolution in the way people thought of cell commitment during deve-
lopment. Since then, a lot of research has been done in understanding
the molecular biology of pluripotent stem cells. iPSCs can be generated
from somatic cells of a patient and therefore have the same genome.
Hence, iPSCs have great potential application in medicine, as they can
be utilized in disease modelling, drug screening and cell replacement
therapy.
breakthrough in research on pluripotent stem cells and their application in medicine
Guðrún Valdimarsdóttir, Anne Richter
Dept. Biochemistry and Molecular Biology, Faculty of Medicine, BMC, University of Iceland
key words: pluripotent stem cells, differentiation, reprogramming, iPSCs, direct lineage reprogramming.
Correspondence: Guðrún Valdimarsdóttir gudrunva@hi.is
38. Li R, Liang J, Ni S, Zhou T, Qing X, Li H, et al. A mesenchy-
mal-to-epithelial transition initiates and is required for the
nuclear reprogramming of mouse fibroblasts. Cell Stem
Cell 2010; 7: 51-63.
39. Samavarchi-Tehrani P, Golipour A, David L, Sung HK,
Beyer TA, Datti A, et al. Functional genomics reveals a
BMP-driven mesenchymal-to-epithelial transition in the
initiation of somatic cell reprogramming. Cell Stem Cell
2010; 7: 64-77.
40. Polo JM, Hochedlinger K. When fibroblasts MET iPSCs.
Cell Stem Cell 2010; 7: 5-6.
41. Santostefano KE, Hamazaki T, Biel NM, Jin S, Umezawa A,
Terada N. A practical guide to induced pluripotent stem
cell research using patient samples. Lab Invest 2015; 95:
4-13.
42. Terrenoire C, Wang K, Tung KW, Chung WK, Pass RH,
Lu JT, et al. Induced pluripotent stem cells used to reveal
drug actions in a long QT syndrome family with complex
genetics. J Gen Physiol 2013; 141: 61-72.
43. Musunuru K. Personalized genomes and cardiovascular
disease. Cold Spring Harb Perspect Med 2015; 5: a014068.
44. Reinhardt P, Schmid B, Burbulla LF, Schondorf DC,
Wagner L, Glatza M, et al. Genetic correction of a LRRK2
mutation in human iPSCs links parkinsonian neurodege-
neration to ERK-dependent changes in gene expression.
Cell Stem Cell 2013; 12: 354-67.
45. Bellin M, Casini S, Davis RP, D'Aniello C, Haas J, Ward-
van Oostwaard D, et al. Isogenic human pluripotent stem
cell pairs reveal the role of a KCNH2 mutation in long-QT
syndrome. Embo J 2013; 32: 3161-75.
46. Cyranoski D. Japanese woman is first recipient of next-
generation stem cells 2014. 15.03.2015; nature.com/news/
japanese-woman-is-first-recipient-of-next-generation-
stem-cells-1.15915 - apríl 2015.
47. Pilot safety study of iPSC-based intervention for wet-
type AMD 2013. 12.03.2015; riken-ibri.jp/AMD/english/
research/index.html - apríl 2015.
48. Moretti A, Laugwitz KL, Dorn T, Sinnecker D, Mummery
C. Pluripotent stem cell models of human heart disease.
Cold Spring Harb Perspect Med 2013; 3: 49.
49. Li HL, Fujimoto N, Sasakawa N, Shirai S, Ohkame T,
Sakuma T, et al. Precise Correction of the Dystrophin
Gene in Duchenne Muscular Dystrophy Patient Induced
Pluripotent Stem Cells by TALEN and CRISPR-Cas9. Stem
Cell Reports 2015; 4: 143-54.
50. Fu Y, Sander JD, Reyon D, Cascio VM, Joung JK.
Improving CRISPR-Cas nuclease specificity using trunca-
ted guide RNAs. Nat Biotechnol 2014; 32: 279-84.
51. Ran FA, Hsu PD, Wright J, Agarwala V, Scott DA, Zhang F.
Genome engineering using the CRISPR-Cas9 system. Nat
Protoc 2013; 8: 2281-308.
52. Tsai SQ, Wyvekens N, Khayter C, Foden JA, Thapar V,
Reyon D, et al. Dimeric CRISPR RNA-guided FokI nuc-
leases for highly specific genome editing. Nat Biotechnol
2014; 32: 569-76.
53. Davis RL, Weintraub H, Lassar AB. Expression of a single
transfected cDNA converts fibroblasts to myoblasts. Cell
1987; 51: 987-1000.
54. Zhou Q, Brown J, Kanarek A, Rajagopal J, Melton DA. In
vivo reprogramming of adult pancreatic exocrine cells to
beta-cells. Nature 2008; 455: 627-32.
55. Islas JF, Liu Y, Weng KC, Robertson MJ, Zhang S, Prejusa
A, et al. Transcription factors ETS2 and MESP1 trans-
differentiate human dermal fibroblasts into cardiac
progenitors. Proc Natl Acad Sci U S A 2012; 109: 13016-21.
56. Efe JA, Hilcove S, Kim J, Zhou H, Ouyang K, Wang G, et
al. Conversion of mouse fibroblasts into cardiomyocytes
using a direct reprogramming strategy. Nat Cell Biol 2011;
13: 215-22.
57. Qian L, Huang Y, Spencer CI, Foley A, Vedantham V,
Liu L, et al. In vivo reprogramming of murine cardiac
fibroblasts into induced cardiomyocytes. Nature 2012; 485:
593-8.
58. Song K, Nam YJ, Luo X, Qi X, Tan W, Huang GN, et al.
Heart repair by reprogramming non-myocytes with
cardiac transcription factors. Nature 2012; 485: 599-604.
Y F I R L I T S G R E I N
Til meðferðar við miðlungsalvarlegum til alvarlegum
þrymlabólum (acne vulgaris).
NÝTT LYF
Lymecycline
Actavis 300 mg
Lymecycline Actavis 300 mg
(Hvert hylki inniheldur 408 mg af lymecýklíni sem jafngildir 300 mg af tetracýklíni.)
Lymecycline Actavis 300 mg, hart hylki. Virkt innihaldsefni: Hvert hylki inniheldur 408 mg af lymecýklíni sem jafngildir 300 mg af tetracýklíni. Ábendingar:
Lymecýklín er ætlað til meðferðar við miðlungsalvarlegum til alvarlegum þrymlabólum (acne vulgaris). Hafa skal í huga opinberar leiðbeiningar um viðeigandi notkun
sýklalyfja. Skammtar og lyfjagjöf: Fullorðnir: Venjulegur skammtur við langtímameðferð gegn miðlungsalvarlegum til alvarlegum þrymlabólum er 1 hylki á dag tekið
með a.m.k. hálfu glasi af vatni í uppréttri stöðu. Hylkið skal taka með léttri máltíð án mjólkurafurða. Meðferð skal haldið áfram í a.m.k. 8 vikur til 12 vikur, en hins vegar
er mikilvægt að takmarka notkun sýklalyfja við eins stutt tímabil og hægt er og hætta notkun þeirra þegar frekari bati er ólíklegur. Meðferð skal ekki haldið áfram í
meira en 6 mánuði. Aldraðir: Eins og á við um önnur tetracýklín er ekki þörf á sértækri skammtaaðlögun.Börn: Notkun er ekki ráðlögð hjá börnum yngri en 12 ára.
Börnum eldri en 12 ára má gefa fullorðinsskammt. Skert nýrnastarfsemi: Útskilnaðarhraði tetracýklíns minnkar þegar um skerta nýrnastarfsemi er að ræða og geta
venjulegir skammtar þannig valdið uppsöfnun. Ef um skerta nýrnastarfsemi er að ræða er ráðlagt að minnka skammtinn og hugsanlega að hafa eftirlit með þéttni í
sermi. Frábendingar: Lymecycline Actavis má ekki nota þegar um er að ræða ofnæmi fyrir lymecýklíni eða öðrum tetracýklínum eða einhverju hjálparefnanna,
sjúklinga með alvarlega skerðingu á nýrnastarfsemi, börn yngri en 12 ára, meðgöngu og brjóstagjöf, samhliðameðferð með retínóíðum til inntöku og notkun er tengist
altækum retínóíðum. Upplýsingar um aukaverkanir, milliverkanir, varnaðarorð og önnur mikilvæg atriði má nálgast í sérlyfjaskrá – www.serlyfjaskra.is.
Pakkningar og hámarksverð í smásölu (september 2015): 300 mg, 100 stk: 7.366 kr. Afgreiðsluflokkur: R. Greiðsluþátttaka: 0. Markaðsleyfishafi: Actavis Group PTC ehf.
Frekari upplýsingar: www.actavis.is, s: 550 3300. Dagsetning síðustu samantektar um eiginleika lyfsins: 10. febrúar 2015. September 2015.
H
V
ÍT
A
H
Ú
S
IÐ
/
S
ÍA
/
A
ct
av
is
5
1
9
0
1
2