Human HPSE2 gene transfer ameliorates bladder pathophysiology in a mutant mouse model of urofacial syndrome

Rare early-onset lower urinary tract disorders include defects of functional maturation of the bladder. Current treatments do not target the primary pathobiology of these diseases. Some have a monogenic basis, such as urofacial, or Ochoa, syndrome (UFS). Here, the bladder does not empty fully becaus...

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Autors principals:	Filipa M Lopes, Celine Grenier, Benjamin W Jarvis, Sara Al Mahdy, Adrian Lène-McKay, Alison M Gurney, William G Newman, Simon N Waddington, Adrian S Woolf, Neil A Roberts
Format:	Artigo
Idioma:	Inglês
Publicat:	eLife Sciences Publications Ltd 2024-07-01
Col·lecció:	eLife
Matèries:	adeno-associated viral vector gene therapy HPSE2 lower urinary tract urinary bladder heparanase 2
Accés en línia:	https://elifesciences.org/articles/91828
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