Concept

Gene therapy

Related publications (480)

Synthetic 3D scaffolds for cancer immunotherapy

Recent clinical success of systemic cancer immunotherapy has paved the way for the next-generation therapeutics. Nevertheless, cancer immunotherapies, in particular combination therapies, are associated in some cases with severe side effects and low respon ...

2019

Non-covalent albumin-binding ligands for extending the circulating half-life of small biotherapeutics

Alessandro Angelini, Alessandro Zorzi, Sara Linciano

Peptides and small protein scaffolds are gaining increasing interest as therapeutics. Similarly to full-length antibodies, they can bind a target with a high binding affinity and specificity while remaining small enough to diffuse into tissues. However, de ...

ROYAL SOC CHEMISTRY2019

Autologous Cell Therapy Approach for Duchenne Muscular Dystrophy using PiggyBac Transposons and Mesoangioblasts

Nicolas Mermod

Duchenne muscular dystrophy (DMD) is a lethal muscle-wasting disease currently without cure. We investigated the use of the PiggyBac transposon for full-length dystrophin expression in murine mesoangioblast (MABs) progenitor cells. DMD murine MABs were tra ...

CELL PRESS2018

Preservation of visual function in an animal model of retinal degeneration using CRISPR/Cas9 gene editing

Diego Ghezzi, Paola Vagni, Tommaso Marchetti

Aims: Inherited Retinal Disorders represent a difficult target for gene therapy. This study exploits the possibility of using CRISPR/Cas9-based gene editing in photoreceptor progenitors to prevent cell death. We performed all the experiments on Rd10 mice, ...

2018

Combinatorial targeting of angiogenesis with inducers of autophagy in the treatment of glioblastoma multiforme

Julie Laetitia Scotton

Malignant gliomas represent 80% of tumors developing in the central nervous system, with 50% being glioblastomas (GBM), the most aggressive form of the disease. The benefit from current therapies is very limited, the overall 5-year survival rate of patient ...

EPFL2018

Engineered anti-CRISPR proteins for optogenetic control of CRISPR-Cas9

Bruno Emanuel Ferreira De Sousa Correia, Zander Harteveld

Anti-CRISPR proteins are powerful tools for CRISPR-Cas9 regulation; the ability to precisely modulate their activity could facilitate spatiotemporally confined genome perturbations and uncover fundamental aspects of CRISPR biology. We engineered optogeneti ...

NATURE PUBLISHING GROUP2018

Pathogenic commonalities between spinal muscular atrophy and amyotrophic lateral sclerosis: Converging roads to therapeutic development

Bernard Schneider

Spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS) are the two most common motoneuron disorders, which share typical pathological hallmarks while remaining genetically distinct. Indeed, SMA is caused by deletions or mutations in the surv ...

ELSEVIER SCIENCE BV2018

Immunoengineering with biomaterials for enhanced cancer immunotherapy

Li Tang, Lixia Wei, Yuqing Xie

Cancer immunotherapy has recently shown dramatic clinical success inducing durable response in patients of a wide variety of malignancies. Further improvement of the clinical outcome with immune related cancer treatment requests more exquisite manipulation ...

2018

Improvement of breast cancer diagnosis through microfluidics

Huu Tuan Nguyen

In breast cancer, 15-20% of cases are reported with overexpression of human epidermal growth factor receptor 2 (HER2) that causes rapid cancer progression and poor prognosis. Fortunately, HER2-targeted therapy using specific antibodies such as Trastuzumab ...

EPFL2018

Genomics: a Swiss army knife to fight leprosy

Charlotte Avanzi

Leprosy, a highly disabling and stigmatizing infectious disease, is caused by Mycobacterium leprae and the newly discovered agent, Mycobacterium lepromatosis. Though treatable with antibiotics, leprosy has still not been eradicated, and around 200,000 new ...

EPFL2018

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