Klinik für Kinder- und Jugendmedizin
Kinderheilkunde I
Allgemeine Pädiatrie, Hämatologie und Onkologie

• Dr. Justin Antony Selvaraj (Junior Group Leader)
  Hematopoietic stem cell gene therapy with Cas9 mRNA and AAV6 vector in metachromatic leukodystrophy
• Andrés Lamsfus Calle (cand. Dr. rer. nat.)
  Gene correction of human β-thalassemic hematopoietic stem cells using CRISPR/Cas9 mechanism
• Alberto Daniel Moreno (cand. Dr. rer. nat.)
  Comparative analysis of lentiviral gene transfer approaches designed to promote fetal hemoglobin production for the treatment of β-hemoglobinopathies
• Guillermo Ureña Bailén (cand. Dr. rer. nat.) CRISPR/Cas9-boosted improvements in CAR-T and CAR-NK cell-based immunotherapy for cancer treatment
• Jennifer Rottenberger (MTA)
  Antibody optimization in CHO-S cells using CRISPR/Cas9 gene editing for enhanced anti-tumor activity
  Safety and Tolerability of donor-specific Mesenchymal Stem Cells in Pediatric Liver Transplantation by Living Donation (Cooperation with Dr. Hartleiff, Tübingen)
• Yujuan Hou (cand. MD PhD) Using CRISPR/Cas9 system for the treatment of immunodeficiencies
• Janani Raju (cand. Dr. rer. nat., funded by Prof. Schöls / Dr. Hayer, Klinik für Neurologie Tübingen) Towards CRISPR/Cas-based gene therapy in adult-onset leukoencephalopathy with axonal spheroids and pigmented glia: generating the optimal guide RNA
• Luise Luib (cand. Dr. med.)
  Etablierung eines Verfahrens zur Früherkennung von ALL-Rezidiven mittels MRD-Bestimmung durch digitale PCR bei Patienten nach anti-CD19-Antikörpertherapie
• Marie Katz (cand. Dr. med.)
  Lack of association between the presence of SUFU polymorphism rs17114808 and development of acute GvHD after hematopoietic stem cell transplantation

• Laura Eichler (cand. Dr. med. 2020)
  Retrospektive Analyse zur Untersuchung der Bedeutung von genetischen Markern in Chitinasegenen bei der Zystischen Fibrose
• Guillermo Ureña Bailén (Master Mol. Med. 2019)
  Generation of efficient CAR-T cells using CRISPR/Cas9 system
• Janani Raju (Master Mol. Med. 2019)
  Optimization of CRISPR/Cas9 system for autologous hematopoietic stem cell transplantation
• Merve Kaftancıoğlu (Master Mol. Med. 2019)
  Hematopoietic stem cell gene therapy with Cas9 mRNA and AAV6 vector in metachromatic leukodystrophy
• Darina Siegmund (Dr. rer. nat. 2018)
  Functional assessment of myeloid-derived suppressor cells, mesenchymal stromal cells, and regulatory T cells for the control of T-cell function: implications for graft-versus-host disease
• Matthias Firnkorn (Dr. med. 2018)
  Zusammenhang zwischen dem KIR-Rezeptorprofil und dem Auftreten akuter lymphoblastischer Leukämien im Kindesalter
• Sibylle Schmidt (Dr. med. 2016)
  Der Einfluss der SNPs rs4077515 des CARD9-Gens und rs3816527 des PTX3-Gens auf die Lungenerkrankung der Zystische Fibrose
• Johanna Huber (Dr. med. 2015)
  Die Bedeutung der KIR-Rezeptoren bei Morbus Crohn und Colitis ulcerosa
• Sebastian Michaelis (Dr. med. 2015)
  Die klinische Relevanz der KIR Rezeptoren bei haploidenter hämatopoietischer Stammzelltransplantation
• Katrin Mörth (Dr. med. 2015)
  Mesenchymale Stromazellen als Therapie bei steroidrefraktärer akuter und chronischer Graft-versus-Host-Disease bei pädiatrischen Patienten nach hämatopoetischer Stammzelltransplantation

• Fortüne Programm Tübingen
• Clinician Scientist Programm Tübingen
• Deutsche José Carreras Leukämie-Stiftung e. V.
• Förderverein für krebskranke Kinder Tübingen e.V.
• Manchot Stiftung
• DEBRA Austria
• Bundesministerium für Bildung und Forschung
• Deutsches Zentrum für Infektionsforschung

• Lamsfus-Calle A*, Daniel-Moreno A*, Antony JS, Epting T, Heumos L, Baskaran P, Admar J, Casadei N, Latifi N, Siegmund D, Kormann M, Handgretinger R, Mezger M (2020) Comparative targeting analysis of KLF1, BCL11A and HBG1/2 in CD34+ HSPCs by CRISPR/Cas9 for the induction of fetal hemoglobin. Nature Scientific Reports 10(1):10133. doi: 10.1038/s41598-020-66309-x.
• Daniel-Moreno A*, Lamsfus-Calle*, Wilber A, Chambers CB, Johnston I, Antony JS, Epting T, Handgretinger R, Mezger M (2020) Comparative analysis of lentiviral gene transfer approaches designed to promote fetal hemoglobin production for the treatment of β-hemoglobinopathies. Blood Cells, Molecules, and Diseases 84: 102456. doi: 10.1016/j.bcmd.2020.102456
• Lamsfus-Calle A*, Daniel-Moreno A*, Ureña-Bailén G, Raju J, Antony J, Handgretinger R, Mezger M (2019) Hematopoietic Stem Cell Gene Therapy: The optimal use of Lentivirus and Gene Editing Approaches. Blood Reviews 15:100641. doi: 10.1016/j.blre.2019.100641
• Oevermann L*, Michaelis S*, Mezger M*, Lang P, Toporski J, Bertaina A, Zecca M, Moretta L, Locatelli F*, Handgretinger R* (2014) KIR B haplotype donors confer a reduced risk of relapse after haploidentical transplantation in children with acute lymphoblastic leukemia. Blood 124: 2744-2747
• Mezger M, Steffens M, Beyer M, Manger C, Eberle J, Toliat MR, Wienker TF, Ljungman P, Hebart H, Dornbusch HJ, Einsele H, Loeffler J (2008) Polymorphisms in the chemokine (C-X-C motif) ligand 10 are associated with invasive aspergillosis after allogeneic stem-cell transplantation and influence CXCL10 expression in monocyte-derived dendritic cells. Blood 111: 534-536

* These authors contributed equally to this work, respectively.