Generation of a gene-corrected isogenic control iPSC line from cystic fibrosis patient-specific iPSCs homozygous for p.Phe508del mutation mediated by TALENs and ssODN

to connected objects

Abstract: Cystic fibrosis (CF) is a monogenetic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which affects multiple organs. Human induced pluripotent stem cells (iPSCs) derived from CF patients and the generation of isogeneic gene-corrected control cell lines enable disease modelling, drug discovery or toxicological studies and therefore the development of CF patient-specific therapies. We have previously generated a hiPSC line from a CF patient homozygous for the p.Phe508del mutation. Here we used TALENs and single-stranded oligonucleotides to correct the mutated triplet in our CF-iPSC line

Location: Deutsche Nationalbibliothek Frankfurt am Main

Extent: Online-Ressource

Language: Englisch

Notes: Stem cell research. - 23 (2017) , 95-97, ISSN: 1876-7753

Event: Veröffentlichung

(where): Freiburg

(who): Universität

(when): 2023

Creator: Merkert, Sylvia
Bednarski, Christien
Göhring, Gudrun
Cathomen, Anton
Martin, Ulrich

Contributor: Centrum für Chronische Immundefizienz

DOI: 10.1016/j.scr.2017.07.010

URN: urn:nbn:de:bsz:25-freidok-1431723

Rights: Open Access; Der Zugriff auf das Objekt ist unbeschränkt möglich.

Last update: 25.03.2025, 1:44 PM CET

Data provider

This object is provided by:
Deutsche Nationalbibliothek. If you have any questions about the object, please contact the data provider.

Show original at data provider

Associated

Time of origin

2023

Other Objects (12)

Hochschulschrift

Establishing a zinc finger nuclease platform for targeted genome editing in human cell lines, primary keratinocyte stem cells and cardiomyocytes in vivo

Hochschulschrift

Entwicklung von gentechnologischen Strategien zur Überwindung der Bluthirnschranke am Modell des Morbus Gaucher Typ 2

Targeted integration of a super-exon into the CFTR locus leads to functional correction of a cystic fibrosis cell line model

TALEN-mediated functional correction of human iPSC-derived macrophages in context of hereditary pulmonary alveolar proteinosis

Generation of safe CAR T cells to target B cell malignancies

Clinical genome editing to treat sickle cell disease - a brief update

Targeted gene transfer to enhance safety and efficacy of immunotherapy

Aufsatzsammlung

CRISPR/Cas9 - einschneidende Revolution in der Gentechnik

Improved specificity and safety of anti-hepatitis B virus TALENs using obligate heterodimeric fokI nuclease domains

TALENs facilitate targeted genome editing in human cells with high specificity and low cytotoxicity

Tracing the specificity of CRISPR-Cas nucleases in clinically relevant human cells

Pre-clinical development of HIV resistant genome edited human CD4+ T cells

Hochschulschrift

Establishing a zinc finger nuclease platform for targeted genome editing in human cell lines, primary keratinocyte stem cells and cardiomyocytes in vivo

Hochschulschrift

Entwicklung von gentechnologischen Strategien zur Überwindung der Bluthirnschranke am Modell des Morbus Gaucher Typ 2

Targeted integration of a super-exon into the CFTR locus leads to functional correction of a cystic fibrosis cell line model

TALEN-mediated functional correction of human iPSC-derived macrophages in context of hereditary pulmonary alveolar proteinosis

Generation of safe CAR T cells to target B cell malignancies

Clinical genome editing to treat sickle cell disease - a brief update

Targeted gene transfer to enhance safety and efficacy of immunotherapy

Aufsatzsammlung

CRISPR/Cas9 - einschneidende Revolution in der Gentechnik

Improved specificity and safety of anti-hepatitis B virus TALENs using obligate heterodimeric fokI nuclease domains

TALENs facilitate targeted genome editing in human cells with high specificity and low cytotoxicity

Tracing the specificity of CRISPR-Cas nucleases in clinically relevant human cells

Pre-clinical development of HIV resistant genome edited human CD4+ T cells

Hochschulschrift

Establishing a zinc finger nuclease platform for targeted genome editing in human cell lines, primary keratinocyte stem cells and cardiomyocytes in vivo

Hochschulschrift

Entwicklung von gentechnologischen Strategien zur Überwindung der Bluthirnschranke am Modell des Morbus Gaucher Typ 2

Targeted integration of a super-exon into the CFTR locus leads to functional correction of a cystic fibrosis cell line model

TALEN-mediated functional correction of human iPSC-derived macrophages in context of hereditary pulmonary alveolar proteinosis

Generation of safe CAR T cells to target B cell malignancies

Clinical genome editing to treat sickle cell disease - a brief update

Targeted gene transfer to enhance safety and efficacy of immunotherapy

Aufsatzsammlung

CRISPR/Cas9 - einschneidende Revolution in der Gentechnik

Improved specificity and safety of anti-hepatitis B virus TALENs using obligate heterodimeric fokI nuclease domains

TALENs facilitate targeted genome editing in human cells with high specificity and low cytotoxicity

Tracing the specificity of CRISPR-Cas nucleases in clinically relevant human cells

Pre-clinical development of HIV resistant genome edited human CD4+ T cells

Cultural heritage institutions wishing to register will find more information here.

Fields marked * need to be filled in.

Username*

Please enter your username

Email*

Please enter your email address

Please do not fill this field

First name

Last name

Password*

Please enter your password

Confirm password*

Please enter the same password

I have read the terms of use and the privacy policy for the collection of personal data and accept them. *

This field is required.

I would like to subscribe to the newsletter of the Deutsche Digitale Bibliothek. See newsletter subscription info.

Account created

Your "My DDB" account has been successfully created. Before you can log in to your account, you must click the confirmation link in the message we just sent to the email address you provided.

Generation of a gene-corrected isogenic control iPSC line from cystic fibrosis patient-specific iPSCs homozygous for p.Phe508del mutation mediated by TALENs and ssODN

Object Details

Contributors, Places and Time

Further information

Data provider

Associated

Time of origin

Other Objects (12)

Establishing a zinc finger nuclease platform for targeted genome editing in human cell lines, primary keratinocyte stem cells and cardiomyocytes in vivo

Entwicklung von gentechnologischen Strategien zur Überwindung der Bluthirnschranke am Modell des Morbus Gaucher Typ 2

Targeted integration of a super-exon into the CFTR locus leads to functional correction of a cystic fibrosis cell line model

TALEN-mediated functional correction of human iPSC-derived macrophages in context of hereditary pulmonary alveolar proteinosis

Generation of safe CAR T cells to target B cell malignancies

Clinical genome editing to treat sickle cell disease - a brief update

Targeted gene transfer to enhance safety and efficacy of immunotherapy

CRISPR/Cas9 - einschneidende Revolution in der Gentechnik

Improved specificity and safety of anti-hepatitis B virus TALENs using obligate heterodimeric fokI nuclease domains

TALENs facilitate targeted genome editing in human cells with high specificity and low cytotoxicity

Tracing the specificity of CRISPR-Cas nucleases in clinically relevant human cells

Pre-clinical development of HIV resistant genome edited human CD4+ T cells

Establishing a zinc finger nuclease platform for targeted genome editing in human cell lines, primary keratinocyte stem cells and cardiomyocytes in vivo

Entwicklung von gentechnologischen Strategien zur Überwindung der Bluthirnschranke am Modell des Morbus Gaucher Typ 2

Targeted integration of a super-exon into the CFTR locus leads to functional correction of a cystic fibrosis cell line model

TALEN-mediated functional correction of human iPSC-derived macrophages in context of hereditary pulmonary alveolar proteinosis

Generation of safe CAR T cells to target B cell malignancies

Clinical genome editing to treat sickle cell disease - a brief update

Targeted gene transfer to enhance safety and efficacy of immunotherapy

CRISPR/Cas9 - einschneidende Revolution in der Gentechnik

Improved specificity and safety of anti-hepatitis B virus TALENs using obligate heterodimeric fokI nuclease domains

TALENs facilitate targeted genome editing in human cells with high specificity and low cytotoxicity

Tracing the specificity of CRISPR-Cas nucleases in clinically relevant human cells

Pre-clinical development of HIV resistant genome edited human CD4+ T cells

Establishing a zinc finger nuclease platform for targeted genome editing in human cell lines, primary keratinocyte stem cells and cardiomyocytes in vivo

Entwicklung von gentechnologischen Strategien zur Überwindung der Bluthirnschranke am Modell des Morbus Gaucher Typ 2

Targeted integration of a super-exon into the CFTR locus leads to functional correction of a cystic fibrosis cell line model

TALEN-mediated functional correction of human iPSC-derived macrophages in context of hereditary pulmonary alveolar proteinosis

Generation of safe CAR T cells to target B cell malignancies

Clinical genome editing to treat sickle cell disease - a brief update

Targeted gene transfer to enhance safety and efficacy of immunotherapy

CRISPR/Cas9 - einschneidende Revolution in der Gentechnik

Improved specificity and safety of anti-hepatitis B virus TALENs using obligate heterodimeric fokI nuclease domains

TALENs facilitate targeted genome editing in human cells with high specificity and low cytotoxicity

Tracing the specificity of CRISPR-Cas nucleases in clinically relevant human cells

Pre-clinical development of HIV resistant genome edited human CD4+ T cells

Related objects

Reset password