Martin G. Sauer
A5055400659- Hematopoietic Stem Cell Transplantation
- CAR-T cell therapy research
- Acute Lymphoblastic Leukemia research
- Hemoglobinopathies and Related Disorders
- Immune Cell Function and Interaction
- Virus-based gene therapy research
- Acute Myeloid Leukemia Research
- Immunodeficiency and Autoimmune Disorders
- Immunotherapy and Immune Responses
- Childhood Cancer Survivors' Quality of Life
- CRISPR and Genetic Engineering
- T-cell and B-cell Immunology
- Lymphoma Diagnosis and Treatment
- Renal Transplantation Outcomes and Treatments
- Blood disorders and treatments
- Cytomegalovirus and herpesvirus research
- Neonatal Health and Biochemistry
- Inflammatory Bowel Disease
- Sociology and Education Studies
- Lung Cancer Treatments and Mutations
- RNA Interference and Gene Delivery
- Neurogenetic and Muscular Disorders Research
- Advanced biosensing and bioanalysis techniques
- Prenatal Screening and Diagnostics
- DNA Repair Mechanisms
Medizinische Hochschule Hannover
2016-2025
Children's Hospital of Los Angeles
2019
University of Southern California
2019
University of California, Los Angeles
2019
Texas Children's Hospital
2019
Dana-Farber Cancer Institute
2019
Lurie Children's Hospital
2019
St. Jude Children's Research Hospital
2019
Hochschule Hannover
2009-2015
Gemeinschaftskrankenhaus Havelhöhe
2014
We performed genetic-linkage analysis and candidate-gene sequencing on samples from two unrelated consanguineous families with children who were affected by early-onset inflammatory bowel disease. screened six additional patients colitis for mutations in candidate genes carried out functional assays patients' peripheral-blood mononuclear cells. an allogeneic hematopoietic stem-cell transplantation one patient.
<h3>Background</h3> Immunodysregulation polyendocrinopathy enteropathy x-linked (IPEX) syndrome is a monogenic autoimmune disease caused by <i>FOXP3</i> mutations. Because it rare disease, the natural history and response to treatments, including allogeneic hematopoietic stem cell transplantation (HSCT) immunosuppression (IS), have not been thoroughly examined. <h3>Objective</h3> This analysis sought evaluate onset, progression, long-term outcome of 2 main treatments in IPEX survivors....
Betibeglogene autotemcel (beti-cel) gene therapy for transfusion-dependent β-thalassemia contains autologous CD34+ hematopoietic stem cells and progenitor transduced with the BB305 lentiviral vector encoding β-globin (βA-T87Q) gene.In this open-label, phase 3 study, we evaluated efficacy safety of beti-cel in adult pediatric patients a non-β0/β0 genotype. Patients underwent myeloablation busulfan (with doses adjusted on basis pharmacokinetic analysis) received intravenously. The primary end...
Genome editing of allogeneic T cells can provide "off-the-shelf" alternatives to autologous chimeric antigen receptor (CAR) cell therapies. Disruption α chain (TRAC) prevent graft-versus-host disease (GVHD) and removal CD52 (cluster differentiation 52) for a survival advantage in the presence alemtuzumab have previously been investigated using transcription activator-like effector nuclease (TALEN)-mediated knockout. Here, we deployed next-generation CRISPR-Cas9 linked CAR expression...
Patients with precursor B-cell acute lymphoblastic leukemia (pB-ALL) who have relapsed after allogeneic hematopoietic stem cell transplantation (allo-HSCT), more than once, or are resistant upfront a dismal prognosis. CD19-targeted chimeric antigen receptor (CAR) T cells evolved as potent immune therapies. Tisagenlecleucel (Tisa-cel) is commercially available autologous CD19-directed CAR T-cell product. We performed retrospective study inviting all centers in Germany to participate....
Human adenovirus (HAdV) infection after hematopoietic stem cell transplantation (HSCT) is associated with significant morbidity and mortality in children. The optimal surveillance treatment strategies are under discussion. Here, we present data from 238 consecutive pediatric allogeneic HSCT recipients who underwent a single center were included prospective, weekly HAdV DNAemia monitoring program by quantitative PCR. loads >1000 copies/mL detected 15.5% of all patients. Despite low directly...
BACKGROUNDAdoptive transfer of EBV-specific T cells can restore specific immunity in immunocompromised patients with EBV-associated complications.METHODSWe provide results a personalized cell manufacturing program evaluating donor, patient, product, and outcome data. Patient-tailored clinical-grade cytotoxic lymphocyte (EBV-CTL) products from stem donors (SCDs), related third-party (TPDs), or unrelated TPDs the allogeneic donor registry (alloCELL) at Hannover Medical School were manufactured...
The introduction of chimeric antigen receptors (CARs) to augment the anticancer activity immune cells represents one major clinical advances in recent years. This work demonstrates that sorted CAR natural killer (NK) have improved antileukemia compared control NK lack a functional CAR. However, terms viability, effectiveness, risk side effects, and practicality applicability, an important question is whether gene-modified cell lines represent better effector than primary human donor CAR-NK...
Abstract GATA2 deficiency is a heterogeneous multi-system disorder characterized by high risk of developing myelodysplastic syndrome (MDS) and myeloid leukemia. We analyzed the outcome 65 patients reported to registry European Working Group (EWOG) MDS in childhood carrying germline mutation ( mut ) who had undergone hematopoietic stem cell transplantation (HSCT). At 5 years probability overall survival disease-free (DFS) was 75% 70%, respectively. Non-relapse mortality relapse equally...
Background: Detection of minimal residual disease (MRD) is an important predictor patient outcome following treatment B-cell acute lymphoblastic leukemia (ALL); importantly, MRD emerging as a useful tool to detect early relapse, which may fulfill key previously unmet clinical need.Objectives: To evaluate the potential predict morphologic relapse in pediatric and young adult ALL (pALL) patients.Design/Method: Bone marrow (BM) peripheral blood (PB) specimens at screening (pre-tisagenlecleucel...
Abstract Total body irradiation (TBI)-based conditioning is associated with superior leukemia-free survival in children ALL undergoing HSCT. However, the risk for subsequent malignant neoplasms (SMN) remains a significant concern. We analyzed 705 pediatric patients enrolled prospective ALL-SCT-BFM-2003 trial and its registry. Patients >2 years received TBI 12 Gy/etoposide ( n = 558) ≤2 of age or contraindications busulfan/cyclophosphamide/etoposide 110). The 5- 10-year cumulative...
Molecular imaging of cells and cellular processes can be achieved by tagging intracellular targets such as receptors, enzymes, or mRNA. Seeking to visualize the presence specific mRNAs magnetic resonance (MR) imaging, we coupled peptide nucleic acids (PNA) with gadolinium-based MR contrast agents using cell-penetrating peptides for delivery. Antisense mRNA DsRed2 protein was used proof principle. The conjugates were produced continuous solid-phase synthesis followed chelation gadolinium....