TY  - THES
A1  - Höfling, Christine
T1  - Gentherapie bei Fanconi Anämie
T1  - gene therapy in fanconi anemia
N2  - Unter Fanconi Anämie versteht man eine rezessiv vererbbare Multisystem-Erkrankung, die einhergeht mit erhöhter spontaner Chromosomenbrüchigkeit, sowie erhöhter Anfälligkeit für toxische Substanzen, wie Mitomycin C (MMC) oder Diepoxybutan (DEB).Gentherapeutische Versuche scheitern bei FA letztlich daran, dass bei fortgeschrittener aplastischer Anämie (= Knochenmarkversagen) die Gewinnung der zur erfolgreichen Transduktion erforderlichen Mengen an CD34 positiven Stamm-Blutzellen schwierig bis unmöglich ist. Die Fortschritte bei den Fremdspender-Transplantationen lassen erwarten, dass zukünftig nahezu alle FA-Patienten mit dieser Therapieform mit guten Erfolgsaussichten behandelt werden können. Insofern ist auch zu erwarten, dass die Option einer somatischen Gentherapie - trotz vielversprechenden Ergebnissen - zukünftig wieder an Bedeutung verlieren wird.
N2  - Fanconi anemia (FA) is an inherited autosomal recessive disease, which is accompanied by increased spontaneous chromosomal breackage, and increased susceptibility to DNA-crosslinking agents such as mitomycin C (MMC) or diepoxybutane (DEB).There are various treatment options to improve life expectancy and quality of life of Fanconi anemia patients. Therapeutic options include administration of androgens or growth factors, hematopoietic stem cell transplantation (HSCT), but also somatic gene therapy. Gene therapy experiments at FA fail in advanced aplastic anemia (bone marrow failure) because getting the necessary quantities of CD34 positive blood stem cells for successful transduction is difficult, if not impossible. With further improvements, HSCT will become the treatment of choice, even though this does not eliminate the life-long thread of solid tumors in FA-patients.
KW  - Gentherapie
KW  - Fanconi-Anämie
KW  - Deutschland / Stammzellgesetz
KW  - Stammzelltransplantation
KW  - gene therapy
KW  - fanconi anemia
KW  - stem cell transplantation
Y1  - 2007
U6  - http://nbn-resolving.de/urn/resolver.pl?urn:nbn:de:bvb:20-opus-26363
ER  - 
TY  - JOUR
A1  - Heil, Hannah S.
A1  - Schreiber, Benjamin
A1  - Götz, Ralph
A1  - Emmerling, Monika
A1  - Dabauvalle, Marie-Christine
A1  - Krohne, Georg
A1  - Höfling, Sven
A1  - Kamp, Martin
A1  - Sauer, Markus
A1  - Heinze, Katrin G.
T1  - Sharpening emitter localization in front of a tuned mirror
JF  - Light: Science & Applications
N2  - Single-molecule localization microscopy (SMLM) aims for maximized precision and a high signal-to-noise ratio1. Both features can be provided by placing the emitter in front of a metal-dielectric nanocoating that acts as a tuned mirror2,3,4. Here, we demonstrate that a higher photon yield at a lower background on biocompatible metal-dielectric nanocoatings substantially improves SMLM performance and increases the localization precision by up to a factor of two. The resolution improvement relies solely on easy-to-fabricate nanocoatings on standard glass coverslips and is spectrally and spatially tunable by the layer design and wavelength, as experimentally demonstrated for dual-color SMLM in cells.
KW  - imaging and sensing
KW  - super-resolution microscopy
Y1  - 2018
U6  - http://nbn-resolving.de/urn/resolver.pl?urn:nbn:de:bvb:20-opus-228080
VL  - 7
ER  -