NicolaBrunetti-PierriMD

Clinical Biochemical Genetics, Clinical Genetics, Pediatric Medical Genetics

Associate Professor

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Office
2 Greenway Plz
Ste 900
Houston, TX 77046
Phone+1 713-798-1750

Fax+1 713-798-1144

Use of Phenylbutyrate Therapy for Patients With Pyruvate Dehydrogenase Complex Deficiency. Start of enrollment: 2018 Oct 01
Completed
Phase 2
Gene Therapy for Severe Crigler Najjar Syndrome Start of enrollment: 2018 Mar 19
Recruiting
Not Applicable
Roles: Principal Investigator

PubMed

Multi-year enzyme expression in patients with mucopolysaccharidosis type VI after liver-directed gene therapy.
Alessandro Rossi, Roberta Romano, Simona Fecarotta, Margherita Dell'Anno, Valentina Pecorella
Med. 2024-11-12
2 citations
Biomarkers for gene therapy clinical trials of lysosomal storage disorders.
Alessandro Rossi, Sabrina Malvagia, Giancarlo la Marca, Giancarlo Parenti, Nicola Brunetti-Pierri
Molecular Therapy. 2024-09-04
2 citations
Vision on gyrate atrophy: why treat the liver?
Iolanda Boffa, Nicola Brunetti-Pierri
EMBO Molecular Medicine. 2024-01-01

Adeno Associated Virus (AAV) Gene Therapy For Mucopolysaccharidosis Type IINational Center For Research Resources2010–2011
Hdad-Mediated Gene Therapy For Hemophilia BNational Heart, Lung, And Blood Institute2009
Adeno Associated Virus (AAV) Gene Therapy For Mucopolysaccharidosis Type IINational Center For Research Resources2008–2009
Hdad-Mediated Gene Therapy For Hemophilia BNational Heart, Lung, And Blood Institute2007–2008