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Emerging gene therapeutics for epidermolysis bullosa under development
The monogenetic disease epidermolysis bullosa (EB) is characterised by the formation of
extended blisters and lesions on the patient's skin upon minimal mechanical stress. Causal …
extended blisters and lesions on the patient's skin upon minimal mechanical stress. Causal …
Heterogeneity of reported outcomes in epidermolysis bullosa clinical research: a sco** review as a first step towards outcome harmonization
EWH Korte, T Welponer, J Kottner… - British Journal of …, 2023 - academic.oup.com
Background Epidermolysis bullosa (EB) is a rare, genetically and clinically heterogeneous
group of skin fragility disorders. No cure is currently available, but many novel and …
group of skin fragility disorders. No cure is currently available, but many novel and …
A scalable and cGMP-compatible autologous organotypic cell therapy for Dystrophic Epidermolysis Bullosa
G Neumayer, JL Torkelson, S Li, K McCarthy… - Nature …, 2024 - nature.com
Abstract We present Dystrophic Epidermolysis Bullosa Cell Therapy (DEBCT), a scalable
platform producing autologous organotypic iPS cell-derived induced skin composite (iSC) …
platform producing autologous organotypic iPS cell-derived induced skin composite (iSC) …
Paired nicking-mediated COL17A1 reframing for junctional epidermolysis bullosa
J Bischof, OP March, B Liemberger, SA Haas, S Hainzl… - Molecular Therapy, 2022 - cell.com
Junctional epidermolysis bullosa (JEB) is a debilitating hereditary skin disorder caused by
mutations in genes encoding laminin-332, type XVII collagen (C17), and integrin-α6β4 …
mutations in genes encoding laminin-332, type XVII collagen (C17), and integrin-α6β4 …
Highly efficient CRISPR/Cas9‐mediated exon skip** for recessive dystrophic epidermolysis bullosa
Gene therapy based on the CRISPR/Cas9 system has emerged as a promising strategy for
treating the monogenic fragile skin disorder recessive dystrophic epidermolysis bullosa …
treating the monogenic fragile skin disorder recessive dystrophic epidermolysis bullosa …
Banana fruit (Musa sp.) DNA-magnetite nanoparticles: Synthesis, characterization, and biocompatibility assays on normal and cancerous cells
D Arregui-Almeida, M Coronel, K Analuisa… - Plos one, 2024 - journals.plos.org
Magnet-mediated gene therapy has gained considerable interest from researchers as a
novel alternative for treating genetic disorders, particularly through the use of …
novel alternative for treating genetic disorders, particularly through the use of …
Enhanced HDR-Mediated Correction of Heterozygous COL7A1 Mutations for Recessive Dystrophic Epidermolysis Bullosa
Gene editing facilitated by homology-directed repair (HDR) holds great potential for treating
monogenetic disorders such as recessive dystrophic epidermolysis bullosa (RDEB) …
monogenetic disorders such as recessive dystrophic epidermolysis bullosa (RDEB) …
Current treatment landscape for dystrophic epidermolysis bullosa: from surgical management to emerging gene therapies and novel skin grafts
Epidermolysis bullosa is a genetic skin disorder characterized by blister formation from
mechanical trauma. Dystrophic epidermolysis bullosa (DEB) is caused by mutations in the …
mechanical trauma. Dystrophic epidermolysis bullosa (DEB) is caused by mutations in the …
Epidermolysis bullosa
Epidermolysis bullosa (EB) is a heterogeneous group of genodermatoses characterized by
mucocutaneous blistering, resulting from intraepidermal or dermo-epidermal tissue …
mucocutaneous blistering, resulting from intraepidermal or dermo-epidermal tissue …
A novel fluorescence-based screen of gene editing molecules for junctional epidermolysis bullosa
J Zwicklhuber, T Kocher, B Liemberger… - International Journal of …, 2023 - mdpi.com
Junctional epidermolysis bullosa (JEB) is a severe blistering skin disease caused by
mutations in genes encoding structural proteins essential for skin integrity. In this study, we …
mutations in genes encoding structural proteins essential for skin integrity. In this study, we …