Dana-farber-cancer Institute Inc.

Children's Medical Center Corporation

Politecnico Di Milano

Ospedale San Raffaele S.r.l.

Fondazione Telethon

Fondazione Telethon Ets

Alessandra Biffi

Davide Moscatelli

Umberto Capasso Palmiero

Marco Peviani

Eleonora Cavalca

Letterio Salvatore Politi

Alessia Capotondo

Renato Auriemma

Rita Milazzo

Mattia Sponchioni

The present invention provides compositions and methods for targeting cells for therapeutic and/or diagnostic purposes, e.g., delivery of therapeutic and/or diagnostic agents to a cell. Nanoparticles and polymers functionalized with capture molecules, reporter molecules, and/or therapeutic agents are provided for the treatment or prevention of disease, including neurological diseases associated with neuroinflammation, and cancer.

Compositions and methods for targeting cells

The invention features compositions and methods for the treatment and prevention of peroxisomal diseases (e.g., neonatal adrenoleukodystrophy and Zellweger syndrome), including in a subject selected as having increased levels of metallothionein polypeptides. The invention also provides compositions and methods for identifying a subject having a peroxisomal disease involving detecting metallothionein polypeptides or polynucleotides.

Compositions and methods for diagnosing and treating peroxisomal diseases

The present invention provides compositions and methods for the treatment or prevention of a neurological disease or disorder of the central nervous system (e.g., a storage disorder, lysosomal storage disorder, neurodegenerative disease, etc.) by reconstitution of brain myeloid cell and microglia upon transplantation of hematopoietic cells enriched in microglia reconstitution potential. The invention also provides compositions and methods for ablating and reconstituting microglia.

Compositions and methods for treating diseases and disorders of the central nervous system

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Boston, MA, United States of America

Alessandra Biffi