首页|Therapeutic targeting of cellular prion protein:toward the development of dual mechanism anti-prion compounds

Therapeutic targeting of cellular prion protein:toward the development of dual mechanism anti-prion compounds

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PrPSc,a misfolded,aggregation-prone isoform of the cellular prion protein(PrPc),is the infectious prion agent responsible for fatal neurodegenerative diseases of humans and other mammals.PrPSc can adopt different pathogenic conformations(prion strains),which can be resistant to potential drugs,or acquire drug resistance,posing challenges for the development of effective therapies.Since PrPc is the obligate precursor of any prion strain and serves as the mediator of prion neurotoxicity,it represents an attractive therapeutic target for prion diseases.In this minireview,we briefly outline the approaches to target PrPc and discuss our recent identification of Zn(Ⅱ)-BnPyP,a PrPc-targeting porphyrin with an unprecedented bimodal mechanism of action.We argue that in-depth understanding of the molecular mechanism by which Zn(Ⅱ)-BnPyP targets PrPc may lead toward the development of a new class of dual mechanism anti-prion compounds.

anti-prion druganti-PrPc antibodyantisense oligonucleotideneurodegenerationpharmacological chaperoneporphyrinprion diseasePrPc degraderPrPc sheddingzinc finger repressor

Antonio Masone、Chiara Zucchelli、Enrico Caruso、Giovanna Musco、Roberto Chiesa

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Laboratory of Prion Neurobiology,Department of Neuroscience,Istituto di Ricerche Farmacologiche Mario Negri IRCCS,Milan,Italy

Biomolecular NMR Unit,Division of Genetics and Cell Biology,IRCCS Ospedale San Raffaele,Milan,Italy

Department of Biotechnology and Life Sciences,University of Insubria,Varese,Italy

2025

中国神经再生研究(英文版)
中国康复医学会

中国神经再生研究(英文版)

影响因子:0.902
ISSN:1673-5374
年,卷(期):2025.20(4)