Cover Image

Gene therapy of the hemoglobinopathies

Sickle cell disease and the ß-thalassemias are caused by mutations of the ß-globin gene and represent the most frequent single gene disorders worldwide. Even in European countries with a previous low frequency of these conditions the prevalence has substantially increased following large scale migra...

Full description

Saved in:
Bibliographic Details
Main Authors: Kunz, Joachim (Author) , Kulozik, Andreas (Author)
Format: Article (Journal)
Language:English
Published: 11 September 2020
In: HemaSphere
Year: 2020, Volume: 4, Issue: 5, Pages: 1-12
ISSN:2572-9241
DOI:10.1097/HS9.0000000000000479
Online Access:Verlag, kostenfrei, Volltext: https://doi.org/10.1097/HS9.0000000000000479
Verlag, kostenfrei, Volltext: https://onlinelibrary.wiley.com/doi/abs/10.1097/HS9.0000000000000479
Get full text
Author Notes:Joachim B. Kunz, Andreas E. Kulozik

MARC

LEADER 00000naa a2200000 c 4500
001 1928857701
003 DE-627
005 20250623094024.0
007 cr uuu---uuuuu
008 250623s2020 xx |||||o 00| ||eng c
024 7 |a 10.1097/HS9.0000000000000479  |2 doi 
035 |a (DE-627)1928857701 
035 |a (DE-599)KXP1928857701 
040 |a DE-627  |b ger  |c DE-627  |e rda 
041 |a eng 
084 |a 33  |2 sdnb 
100 1 |a Kunz, Joachim  |d 1974-  |e VerfasserIn  |0 (DE-588)124297730  |0 (DE-627)085769029  |0 (DE-576)294107703  |4 aut 
245 1 0 |a Gene therapy of the hemoglobinopathies  |c Joachim B. Kunz, Andreas E. Kulozik 
264 1 |c 11 September 2020 
300 |b Illustrationen 
300 |a 12 
336 |a Text  |b txt  |2 rdacontent 
337 |a Computermedien  |b c  |2 rdamedia 
338 |a Online-Ressource  |b cr  |2 rdacarrier 
500 |a Zuerst online veröffentlicht: 11 September 2020 
500 |a Gesehen am 23.06.2025 
520 |a Sickle cell disease and the ß-thalassemias are caused by mutations of the ß-globin gene and represent the most frequent single gene disorders worldwide. Even in European countries with a previous low frequency of these conditions the prevalence has substantially increased following large scale migration from Africa and the Middle East to Europe. The hemoglobin diseases severely limit both, life expectancy and quality of life and require either life-long supportive therapy if cure cannot be achieved by allogeneic stem cell transplantation. Strategies for ex vivo gene therapy aiming at either re-establishing normal ß-globin chain synthesis or at re-activating fetal γ-globin chain and HbF expression are currently in clinical development. The European Medicine Agency (EMA) conditionally licensed gene addition therapy based on lentiviral transduction of hematopoietic stem cells in 2019 for a selected group of patients with transfusion dependent non-ß° thalassemia major without a suitable stem cell donor. Gene therapy thus offers a relevant chance to this group of patients for whom cure has previously not been on the horizon. In this review, we discuss the potential and the challenges of gene addition and gene editing strategies for the hemoglobin diseases. 
700 1 |a Kulozik, Andreas  |d 1959-  |e VerfasserIn  |0 (DE-588)1025736567  |0 (DE-627)723892032  |0 (DE-576)167091638  |4 aut 
773 0 8 |i Enthalten in  |t HemaSphere  |d Hoboken : John Wiley & Sons Ltd., 2017  |g 4(2020), 5, Artikel-ID e479, Seite 1-12  |h Online-Ressource  |w (DE-627)1015324924  |w (DE-600)2922183-3  |w (DE-576)500571066  |x 2572-9241  |7 nnas  |a Gene therapy of the hemoglobinopathies 
773 1 8 |g volume:4  |g year:2020  |g number:5  |g elocationid:e479  |g pages:1-12  |g extent:12  |a Gene therapy of the hemoglobinopathies 
856 4 0 |u https://doi.org/10.1097/HS9.0000000000000479  |x Verlag  |x Resolving-System  |z kostenfrei  |3 Volltext 
856 4 0 |u https://onlinelibrary.wiley.com/doi/abs/10.1097/HS9.0000000000000479  |x Verlag  |z kostenfrei  |3 Volltext 
951 |a AR 
992 |a 20250623 
993 |a Article 
994 |a 2020 
998 |g 1025736567  |a Kulozik, Andreas  |m 1025736567:Kulozik, Andreas  |d 910000  |d 910500  |e 910000PK1025736567  |e 910500PK1025736567  |k 0/910000/  |k 1/910000/910500/  |p 2  |y j 
998 |g 124297730  |a Kunz, Joachim  |m 124297730:Kunz, Joachim  |d 910000  |d 910500  |d 50000  |e 910000PK124297730  |e 910500PK124297730  |e 50000PK124297730  |k 0/910000/  |k 1/910000/910500/  |k 0/50000/  |p 1  |x j 
999 |a KXP-PPN1928857701  |e 4737335843 
BIB |a Y 
SER |a journal 
JSO |a {"title":[{"title":"Gene therapy of the hemoglobinopathies","title_sort":"Gene therapy of the hemoglobinopathies"}],"name":{"displayForm":["Joachim B. Kunz, Andreas E. Kulozik"]},"type":{"media":"Online-Ressource","bibl":"article-journal"},"recId":"1928857701","language":["eng"],"physDesc":[{"noteIll":"Illustrationen","extent":"12 S."}],"person":[{"family":"Kunz","given":"Joachim","display":"Kunz, Joachim","role":"aut","roleDisplay":"VerfasserIn"},{"display":"Kulozik, Andreas","role":"aut","roleDisplay":"VerfasserIn","family":"Kulozik","given":"Andreas"}],"note":["Zuerst online veröffentlicht: 11 September 2020","Gesehen am 23.06.2025"],"origin":[{"dateIssuedKey":"2020","dateIssuedDisp":"11 September 2020"}],"id":{"eki":["1928857701"],"doi":["10.1097/HS9.0000000000000479"]},"relHost":[{"origin":[{"dateIssuedDisp":"2024-","dateIssuedKey":"2024","publisher":"John Wiley & Sons Ltd. ; Wolters Kluwer Health","publisherPlace":"Hoboken ; [Philadelphia, Pennsylvania]"}],"part":{"issue":"5","text":"4(2020), 5, Artikel-ID e479, Seite 1-12","pages":"1-12","volume":"4","extent":"12","year":"2020"},"id":{"zdb":["2922183-3"],"issn":["2572-9241"],"eki":["1015324924"]},"pubHistory":["Volume 1, issue 1 (December 2017)-"],"title":[{"title_sort":"HemaSphere","title":"HemaSphere","subtitle":"open access journal of the European Hematology Association"}],"recId":"1015324924","corporate":[{"role":"isb","roleDisplay":"Herausgebendes Organ","display":"European Hematology Association"}],"language":["eng"],"physDesc":[{"extent":"Online-Ressource"}],"note":["Gesehen am 18.01.2024"],"disp":"Gene therapy of the hemoglobinopathiesHemaSphere","type":{"media":"Online-Ressource","bibl":"periodical"}}]} 
SRT |a KUNZJOACHIGENETHERAP1120 

Similar Items