List of works - Christof von Kalle

Uncovering and dissecting the genotoxicity of self-inactivating lentiviral vectors in vivo

scientific article

Evaluation of TCR Gene Editing Achieved by TALENs, CRISPR/Cas9, and megaTAL Nucleases

scientific article published on 27 October 2015

In Vivo Tracking of Human Hematopoiesis Reveals Patterns of Clonal Dynamics during Early and Steady-State Reconstitution Phases

scientific article published on 25 May 2016

Clonality analysis after retroviral-mediated gene transfer to CD34+ cells from the cord blood of ADA-deficient SCID neonates

scientific article published on 17 March 2003

Methylguanine methyltransferase-mediated in vivo selection and chemoprotection of allogeneic stem cells in a large-animal model

scientific article published on November 2003

Selective survival of peripheral blood lymphocytes in children with HIV-1 following delivery of an anti-HIV gene to bone marrow CD34(+) cells

scientific article

MicroRNA-sensitive oncolytic measles viruses for cancer-specific vector tropism

scientific article

Genome-wide mapping of foamy virus vector integrations into a human cell line

scientific article published on May 2006

Preclinical safety and efficacy of human CD34(+) cells transduced with lentiviral vector for the treatment of Wiskott-Aldrich syndrome

scientific article

Thymus-autonomous T cell development in the absence of progenitor import

scientific article

Long-term clinical and molecular follow-up of large animals receiving retrovirally transduced stem and progenitor cells: no progression to clonal hematopoiesis or leukemia

scientific article published in March 2004

Integration profile of retroviral vector in gene therapy treated patients is cell-specific according to gene expression and chromatin conformation of target cell

scientific article

Lentiviral vector-based insertional mutagenesis identifies genes associated with liver cancer

scientific article published on 13 January 2013

Fanconi anemia gene editing by the CRISPR/Cas9 system

scientific article

Lentiviral hematopoietic cell gene therapy for X-linked adrenoleukodystrophy

scientific article published in January 2012

Failure of SCID-X1 gene therapy in older patients

scientific article

Preclinical evaluation of efficacy and safety of an improved lentiviral vector for the treatment of β-thalassemia and sickle cell disease

scientific article

Pharmacologically regulated in vivo selection in a large animal

scientific article published in September 2002

Lentiviral vector integration profiles differ in rodent postmitotic tissues

scientific article

Genome editing technologies: defining a path to clinic

scientific article published on May 2015

Lentiviral vectors pseudotyped with murine ecotropic envelope: increased biosafety and convenience in preclinical research

scientific article published in May 2006

Extensive methylation of promoter sequences silences lentiviral transgene expression during stem cell differentiation in vivo

scientific article

Artificial riboswitches for gene expression and replication control of DNA and RNA viruses

scientific article published on January 21, 2014

Retroviral Gene Therapy for X-linked Chronic Granulomatous Disease: Results From Phase I/II Trial

scientific article published on August 30, 2011

High-resolution analysis of the human T-cell receptor repertoire

scientific article published in September 2015

Bioinformatic clonality analysis of next-generation sequencing-derived viral vector integration sites

scientific article

Clonal evidence for the transduction of CD34+ cells with lymphomyeloid differentiation potential and self-renewal capacity in the SCID-X1 gene therapy trial

scientific article published on 7 December 2004

Granulocyte-macrophage colony-stimulating factor-armed oncolytic measles virus is an effective therapeutic cancer vaccine

scientific article published on July 2013

Mutations in ROGDI Cause Kohlschütter-Tönz Syndrome

scientific article

Continued response off treatment after BRAF inhibition in refractory hairy cell leukemia

scientific article

In vivo gene transfer into adult stem cells in unconditioned mice by in situ delivery of a lentiviral vector

scientific article

Recurrent CDKN1B (p27) mutations in hairy cell leukemia

scientific article

Integration frequency and intermolecular recombination of rAAV vectors in non-human primate skeletal muscle and liver

scientific article

Stable human FIX expression after 0.9G intrauterine gene transfer of self-complementary adeno-associated viral vector 5 and 8 in macaques

scientific article published on 31 May 2011

Drug-perturbation-based stratification of blood cancer

scientific article published on 11 December 2017

Targeted resequencing for analysis of clonal composition of recurrent gene mutations in chronic lymphocytic leukaemia

scientific article

Insertion sites in engrafted cells cluster within a limited repertoire of genomic areas after gammaretroviral vector gene therapy

scientific article published on 23 August 2011

Lentivirus-mediated reprogramming of somatic cells in the absence of transgenic transcription factors

scientific article

Ex vivo treatment of proliferating human cord blood stem cells with stroma-derived factor-1 enhances their ability to engraft NOD/SCID mice

scientific article

Molecular evidence of lentiviral vector-mediated gene transfer into human self-renewing, multi-potent, long-term NOD/SCID repopulating hematopoietic cells

scientific article published on November 2002

BRAF inhibition in hairy cell leukemia with low-dose vemurafenib

scientific article

Real-time definition of non-randomness in the distribution of genomic events

scientific article published on 27 June 2007

Precision oncology based on omics data: The NCT Heidelberg experience

scientific article

Integration-deficient Lentiviral Vectors Expressing Codon-optimized R338L Human FIX Restore Normal Hemostasis in Hemophilia B Mice

scientific article published on August 14, 2013

Challenges in vector and trial design using retroviral vectors for long-term gene correction in hematopoietic stem cell gene therapy

scientific article published in June 2012

Integration of genomics and histology revises diagnosis and enables effective therapy of refractory cancer of unknown primary with PDL1 amplification

scientific article

High-definition mapping of retroviral integration sites defines the fate of allogeneic T cells after donor lymphocyte infusion

scientific article

The impact of low-dose busulfan on clonal dynamics in nonhuman primates

scientific article

Efficient marking of human cells with rapid but transient repopulating activity in autografted recipients

scientific article published on 21 April 2005

Parallel assessment of globin lentiviral transfer in induced pluripotent stem cells and adult hematopoietic stem cells derived from the same transplanted β-thalassemia patient

scientific article published in September 2013

List of works by Christof von Kalle

Uncovering and dissecting the genotoxicity of self-inactivating lentiviral vectors in vivo

Evaluation of TCR Gene Editing Achieved by TALENs, CRISPR/Cas9, and megaTAL Nucleases

In Vivo Tracking of Human Hematopoiesis Reveals Patterns of Clonal Dynamics during Early and Steady-State Reconstitution Phases

Clonality analysis after retroviral-mediated gene transfer to CD34+ cells from the cord blood of ADA-deficient SCID neonates

Methylguanine methyltransferase-mediated in vivo selection and chemoprotection of allogeneic stem cells in a large-animal model

Selective survival of peripheral blood lymphocytes in children with HIV-1 following delivery of an anti-HIV gene to bone marrow CD34(+) cells

MicroRNA-sensitive oncolytic measles viruses for cancer-specific vector tropism

Genome-wide mapping of foamy virus vector integrations into a human cell line

Preclinical safety and efficacy of human CD34(+) cells transduced with lentiviral vector for the treatment of Wiskott-Aldrich syndrome

Thymus-autonomous T cell development in the absence of progenitor import

Long-term clinical and molecular follow-up of large animals receiving retrovirally transduced stem and progenitor cells: no progression to clonal hematopoiesis or leukemia

Integration profile of retroviral vector in gene therapy treated patients is cell-specific according to gene expression and chromatin conformation of target cell

Lentiviral vector-based insertional mutagenesis identifies genes associated with liver cancer

Fanconi anemia gene editing by the CRISPR/Cas9 system

Lentiviral hematopoietic cell gene therapy for X-linked adrenoleukodystrophy

Failure of SCID-X1 gene therapy in older patients

Preclinical evaluation of efficacy and safety of an improved lentiviral vector for the treatment of β-thalassemia and sickle cell disease

Pharmacologically regulated in vivo selection in a large animal

Lentiviral vector integration profiles differ in rodent postmitotic tissues

Genome editing technologies: defining a path to clinic

Lentiviral vectors pseudotyped with murine ecotropic envelope: increased biosafety and convenience in preclinical research

Extensive methylation of promoter sequences silences lentiviral transgene expression during stem cell differentiation in vivo

Artificial riboswitches for gene expression and replication control of DNA and RNA viruses

Retroviral Gene Therapy for X-linked Chronic Granulomatous Disease: Results From Phase I/II Trial

High-resolution analysis of the human T-cell receptor repertoire

Bioinformatic clonality analysis of next-generation sequencing-derived viral vector integration sites

Clonal evidence for the transduction of CD34+ cells with lymphomyeloid differentiation potential and self-renewal capacity in the SCID-X1 gene therapy trial

Granulocyte-macrophage colony-stimulating factor-armed oncolytic measles virus is an effective therapeutic cancer vaccine

Mutations in ROGDI Cause Kohlschütter-Tönz Syndrome

Continued response off treatment after BRAF inhibition in refractory hairy cell leukemia

In vivo gene transfer into adult stem cells in unconditioned mice by in situ delivery of a lentiviral vector

Recurrent CDKN1B (p27) mutations in hairy cell leukemia

Integration frequency and intermolecular recombination of rAAV vectors in non-human primate skeletal muscle and liver

Stable human FIX expression after 0.9G intrauterine gene transfer of self-complementary adeno-associated viral vector 5 and 8 in macaques

Drug-perturbation-based stratification of blood cancer

Targeted resequencing for analysis of clonal composition of recurrent gene mutations in chronic lymphocytic leukaemia

Insertion sites in engrafted cells cluster within a limited repertoire of genomic areas after gammaretroviral vector gene therapy

Lentivirus-mediated reprogramming of somatic cells in the absence of transgenic transcription factors

Ex vivo treatment of proliferating human cord blood stem cells with stroma-derived factor-1 enhances their ability to engraft NOD/SCID mice

Molecular evidence of lentiviral vector-mediated gene transfer into human self-renewing, multi-potent, long-term NOD/SCID repopulating hematopoietic cells

BRAF inhibition in hairy cell leukemia with low-dose vemurafenib

Real-time definition of non-randomness in the distribution of genomic events

Precision oncology based on omics data: The NCT Heidelberg experience

Integration-deficient Lentiviral Vectors Expressing Codon-optimized R338L Human FIX Restore Normal Hemostasis in Hemophilia B Mice

Challenges in vector and trial design using retroviral vectors for long-term gene correction in hematopoietic stem cell gene therapy

Integration of genomics and histology revises diagnosis and enables effective therapy of refractory cancer of unknown primary with PDL1 amplification

High-definition mapping of retroviral integration sites defines the fate of allogeneic T cells after donor lymphocyte infusion

The impact of low-dose busulfan on clonal dynamics in nonhuman primates

Efficient marking of human cells with rapid but transient repopulating activity in autografted recipients

Parallel assessment of globin lentiviral transfer in induced pluripotent stem cells and adult hematopoietic stem cells derived from the same transplanted β-thalassemia patient