Mirian Farinon

Ms. Mirian Farinon


Research Focus & Metrics

Academic Career Length

10Years (2017-Present)

(Calculated once Scilit publication data is synced: current year − year of first publication + 1)

Representative Publications

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1 March 2017 in European Journal of Pharmacology
Verified
Journal Article
Closed access
MFMirian Farinon
Mirian Farinon
VCVanessa S. Clarimundo
Vanessa S. Clarimundo
GPGraziele P.R. Pedrazza
Graziele P.R. Pedrazza
PGPercio S. S. Gulko
Percio S. S. Gulko
JZJosé Angelo Silveira Zuanazzi
José Angelo Silveira Zuanazzi
POPatricia G. de Oliveira
Patricia G. de Oliveira

24 February 2026 in Pharmaceutics
Verified
Journal Article
Open access
PDF file
HSHallana Souza Santos
Hallana Souza Santos
Edina PolettoEdina Poletto
Edina Poletto
LVLuisa Natalia Pimentel Vera
Luisa Natalia Pimentel Vera
MFMirian Farinon
Mirian Farinon
Francyne KubaskiFrancyne Kubaski
Francyne Kubaski
Guilherme BaldoGuilherme Baldo
Guilherme Baldo
Background/Objectives: Mucopolysaccharidosis type I (MPS I) is a lysosomal storage disorder caused by α-L-iduronidase (IDUA) deficiency, leading to progressive glycosaminoglycan (GAG) accumulation and severe joint involvement. Gene editing represents a promising alternative to restore localized enzyme production. Therefore, this study aimed to evaluate the feasibility, efficacy, and safety of in situ genome editing through intra-articular administration of a nonviral CRISPR/Cas9 system to increase localized IDUA expression in an MPS I mouse model. Methods: Cationic liposomes were formulated to deliver plasmids encoding the CRISPR/Cas9 system targeted to the ROSA26 locus along with an IDUA donor sequence. In vitro assays were performed in fibroblast-like synoviocytes (FLSs) isolated from MPS I mice to assess cytotoxicity, gene editing efficiency, and IDUA activity. In vivo, MPS I mice received intra-articular injections in the knee joints, either as a single dose (short-term study) or monthly for three months (long-term study). IDUA activity, GAG levels, and genome editing efficiency were evaluated in joint tissues, synovial fluid, serum, and major organs. Results: Gene-edited FLS showed sustained IDUA activity for up to 30 days with low cytotoxicity. In vivo, intra-articular administration resulted in a significant increase in IDUA activity in joint tissue and synovial fluid without detectable systemic IDUA. Long-term treatment led to persistent joint-localized IDUA activity, significant reductions (>50%) in GAG levels, and detectable genome editing in joint DNA. Conclusions: Intra-articular delivery of CRISPR/Cas9 via cationic liposomes enables safe and effective localized genome editing, representing a promising strategy for treating joint manifestations of MPS I.