Concept

Thérapie génique

Publications associées (480)

Alpha-Synuclein oligomerization and aggregation: All models are useful but only if we know what they model This is the reply to a comment "Alpha-synuclein oligomerization and aggregation: A model will always be a model" on the original article "Monitoring alpha-synuclein oligomerization and aggregation using bimolecular fluorescence complementation assays: What you see is not always what you get". The articles are accompanied by a Preface "How good are cellular models?".

Alpha-synuclein oligomerization is one of the early events on the pathway to Lewy body formation. Therefore, interfering with this process holds tremendous potential for developing therapies that block alpha-Syn pathology formation and toxicity. The develo ...

WILEY2021

Il10/fc fusion proteins useful as enhancers of immunotherapies

Li Tang, Yugang Guo, Yuqing Xie

The present disclosure relates to new agents useful for anti-cancer therapy such as anti-cancer adoptive T-cell transfer (ACT) immunotherapy or immune check-point blockade therapy and related compositions, uses and methods thereof. ...

2021

Physically asymmetric division of the C. elegans zygote ensures invariably successful embryogenesis

Radek Jankele

Asymmetric divisions that yield daughter cells of different sizes are frequent during early embryogenesis, but the importance of such a physical difference for successful development remains poorly understood. Here, we investigated this question using the ...

ELIFE SCIENCES PUBLICATIONS LTD2021

Gene replacement therapy provides benefit in an adult mouse model of Leigh syndrome

Johan Auwerx

Mutations in nuclear-encoded mitochondrial genes are responsible for a broad spectrum of disorders among which Leigh syndrome is the most common in infancy. No effective therapies are available for this severe disease mainly because of the limited capabili ...

2020

Selection of Water-Soluble Chitosan by Microwave-Assisted Degradation and pH-Controlled Precipitation

Sandrine Gerber, Yann Lavanchy, Céline Marie Anne Journot, Laura Camille Louise Nicolle

In the field of gene therapy, chitosan (CS) gained interest for its promise as a non-viral DNA vector. However, commercial sources of CS lack precise characterization and do not generally reach sufficient solubility in aqueous media for in vitro and in viv ...

2020

Multicomponent Alginate-Derived Hydrogel Microspheres Presenting Hybrid Ionic-Covalent Network and Drug Eluting Properties

Sandrine Gerber, François Rémi Pierre Noverraz, Luca Szabó

The development of multifunctional encapsulation biomaterials could help the translation of cell‐based therapies into standard medical care. One of the major hurdles in the field of encapsulated cell transplantation is the current lack of materials present ...

2020

Targeting the oncogenic signaling of the transcription factor STAT3 with potent and selective monobody inhibitors

Grégory La Sala

Many targeted cancer therapies fail to improve the overall survival of patients. Limitations involve low drug selectivity and the rapid development of resistance. Moreover, the majority of oncogenic drivers presently remain considered as undruggable as th ...

EPFL2020

Single-Cell Tracing Dissects Regulation of Maintenance and Inheritance of Transcriptional Reinduction Memory

Sebastian Maerkl, Johannes Becker

Transcriptional memory of gene expression enables adaptation to repeated stimuli across many organisms. However, the regulation and heritability of transcriptional memory in single cells and through divisions remains poorly understood. Here, we combined mi ...

CELL PRESS2020

Efficient viral transduction in mouse inner ear hair cells with utricle injection and AAV9-PHP.B

Bernard Schneider, Paola Andrea Solanes Vega

Viral delivery of exogenous coding sequences into the inner ear has the potential for therapeutic benefit for patients suffering genetic or acquired hearing loss. To devise improved strategies for viral delivery, we investigated two injection techniques, r ...

2020

A computationally designed chimeric antigen receptor provides a small-molecule safety switch for T-cell therapy

Bruno Emanuel Ferreira De Sousa Correia, George Coukos, Pablo Gainza Cirauqui, Jaume Bonet Martinez, Sabrina Susan Vollers, Sailan Shui

The activity of CAR-T cells is reversibly halted with a small-molecule drug. Approaches to increase the activity of chimeric antigen receptor (CAR)-T cells against solid tumors may also increase the risk of toxicity and other side effects. To improve the s ...

2020

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