Improved double-nicking strategies for COL7A1-editing by homologous recombination
T Kocher, RN Wagner, A Klausegger… - … Therapy-Nucleic Acids, 2019 - cell.com
Current gene-editing approaches for treatment of recessive dystrophic epidermolysis
bullosa (RDEB), an inherited, severe form of blistering skin disease, suffer from low …
bullosa (RDEB), an inherited, severe form of blistering skin disease, suffer from low …
Psychosocial recommendations for the care of children and adults with epidermolysis bullosa and their family: evidence based guidelines
K Martin, S Geuens, JK Asche, R Bodan… - Orphanet journal of rare …, 2019 - Springer
Epidermolysis Bullosa (EB) is a group of rare genetic disorders resulting in skin fragility and
other symptoms. Commissioned by DEBRA International and funded by DEBRA Norway …
other symptoms. Commissioned by DEBRA International and funded by DEBRA Norway …
Squamous cell carcinoma in patients with inherited epidermolysis bullosa: Review of current literature
D Bonamonte, A Filoni, A De Marco, L Lospalluti… - Cells, 2022 - mdpi.com
Epidermolysis bullosa (EB) is a group of rare congenital diseases caused by mutations in
structural proteins of the dermal/epidermal junction that are characterized by extreme …
structural proteins of the dermal/epidermal junction that are characterized by extreme …
Newer treatment modalities in epidermolysis bullosa
L Bruckner-Tuderman - Indian Dermatology Online Journal, 2019 - journals.lww.com
The term epidermolysis bullosa (EB) refers to a group of hereditary skin blistering diseases.
The group is clinically and genetically heterogeneous, but all EB forms are associated with …
The group is clinically and genetically heterogeneous, but all EB forms are associated with …
[HTML][HTML] Predictable CRISPR/Cas9-mediated COL7A1 reframing for dystrophic epidermolysis bullosa
End-joining‒based gene editing is frequently used for efficient reframing and knockout of
target genes. However, the associated random, unpredictable, and often heterogeneous …
target genes. However, the associated random, unpredictable, and often heterogeneous …
Epidermolysis Bullosa in children: the central role of the pediatrician
MR Marchili, G Spina, M Roversi, C Mascolo… - Orphanet journal of rare …, 2022 - Springer
Epidermolysis bullosa (EB) is a severe hereditary disease characterized by defective
epithelial adhesion causing mucocutaneous fragility. The major types are EB simplex (EBS) …
epithelial adhesion causing mucocutaneous fragility. The major types are EB simplex (EBS) …
Hologene 5: a phase II/III clinical trial of combined cell and gene therapy of junctional epidermolysis bullosa
L De Rosa, E Enzo, G Zardi, C Bodemer… - Frontiers in …, 2021 - frontiersin.org
Epidermolysis bullosa (EB) is a group of devastating genetic diseases characterized by skin
and mucosal fragility and formation of blisters, which develop either spontaneously or in …
and mucosal fragility and formation of blisters, which develop either spontaneously or in …
Multidisciplinary care for patients with epidermolysis bullosa from birth to adolescence: experience of one Italian reference center
C Retrosi, A Diociaiuti, C De Ranieri… - Italian Journal of …, 2022 - Springer
Background Epidermolysis bullosa (EB) is a disabling and chronic genodermatosis
characterized by mucocutaneous fragility with blister formation after minimal trauma …
characterized by mucocutaneous fragility with blister formation after minimal trauma …
[HTML][HTML] Dystrophic epidermolysis bullosa: COL7A1 mutation landscape in a multi-ethnic cohort of 152 extended families with high degree of customary …
Dystrophic epidermolysis bullosa is a heritable skin disease manifesting with sub-lamina
densa blistering, erosions, and chronic ulcers. COL7A1, encoding type VII collagen, has …
densa blistering, erosions, and chronic ulcers. COL7A1, encoding type VII collagen, has …
A non-viral and selection-free COL7A1 HDR approach with improved safety profile for dystrophic epidermolysis bullosa
Gene editing via homology-directed repair (HDR) currently comprises the best strategy to
obtain perfect corrections for pathogenic mutations of monogenic diseases, such as the …
obtain perfect corrections for pathogenic mutations of monogenic diseases, such as the …