A5006938194- Cancer Genomics and Diagnostics
- CRISPR and Genetic Engineering
- DNA Repair Mechanisms
- Lung Cancer Treatments and Mutations
- Cancer Treatment and Pharmacology
- RNA and protein synthesis mechanisms
- Single-cell and spatial transcriptomics
- Virus-based gene therapy research
- Advanced biosensing and bioanalysis techniques
- CAR-T cell therapy research
- RNA Interference and Gene Delivery
- Pluripotent Stem Cells Research
- Cancer-related molecular mechanisms research
- Gene Regulatory Network Analysis
- Viral Infectious Diseases and Gene Expression in Insects
- Congenital heart defects research
- Genetics and Neurodevelopmental Disorders
- RNA Research and Splicing
- Gut microbiota and health
- Transgenic Plants and Applications
- Light effects on plants
- Cell Image Analysis Techniques
- MicroRNA in disease regulation
- Photoreceptor and optogenetics research
- Nicotinic Acetylcholine Receptors Study
University of Basel
2017-2024
ETH Zurich
2017-2024
NCCR Chemical Biology - Visualisation and Control of Biological Processes Using Chemistry
2023
Fondation Botnar
2023
Broad Institute
2013-2020
McGovern Institute for Brain Research
2013-2020
Massachusetts Institute of Technology
2013-2020
Harvard University
2017
IIT@MIT
2016
University of Utah
2012-2014
Significance Understanding the design rules that govern structure and function of natural biological systems gives us ability to forward engineer machines integrated with powered by components. Such machines, or “bio-bots,” can sense, process, respond dynamic environmental signals in real time, enabling a variety applications. Here we present modular optogenetic muscle actuator used power actuation locomotion 3D printed flexible skeletons. Observing controlling functional response such...
Autism spectrum disorder (ASD) is a heterogeneous disease, but genetically defined models can provide an entry point to studying the molecular underpinnings of this disorder. We generated germline mutant mice with loss-of-function mutations in Chd8, de novo mutation strongly associated ASD, and demonstrate that these display hallmark ASD behaviors, macrocephaly, craniofacial abnormalities similar patient phenotypes. Chd8+/– broad, brain-region-specific dysregulation major regulatory cellular...
Motor units are the fundamental elements responsible for muscle movement. They formed by lower motor neurons and their targets, synapsed via neuromuscular junctions (NMJs). The loss of NMJs in neurodegenerative disorders (such as amyotrophic lateral sclerosis or spinal atrophy) a result traumatic injuries affects millions lives each year. Developing vitro assays that closely recapitulate physiology tissues is crucial to understand formation maturation NMJs, well help unravel mechanisms...
It is estimated that only 0.02% of disseminated tumour cells are able to seed overt metastases
AAV-mediated CRISPR screens empower autochthonous functional cancer genomics in the mouse liver.
Transcriptional recording by CRISPR spacer acquisition from RNA endows engineered Escherichia coli with synthetic memory, which through Record-seq reveals transcriptome-scale records. Microbial sentinels that traverse the gastrointestinal tract capture a wide range of genes and pathways describe interactions host, including quantitative shifts in molecular environment result alterations host diet, induced inflammation, microbiome complexity. We demonstrate multiplexed using barcoded arrays,...
The ever-growing compendium of genetic variants associated with human pathologies demands new methods to study genotype-phenotype relationships in complex tissues a high-throughput manner1,2. Here we introduce adeno-associated virus (AAV)-mediated direct vivo single-cell CRISPR screening, termed AAV-Perturb-seq, tuneable and broadly applicable method for transcriptional linkage analysis as well high-resolution phenotyping perturbations vivo. We applied AAV-Perturb-seq using gene editing...
Clustered regularly interspaced short palindromic repeats (CRISPR) screens emerged as the gold standard technology in genetic screening recent years. Most CRISPR are conducted vitro, although current technologies fail to completely recapitulate vivo physiological environment. Direct - where cells targeted within their natural niche is emerging a powerful approach unravel biological processes intact tissues and organs, taking into account complex cellular interactions, immune response,...
Mutational scanning connects genetic variants to phenotype, enabling the interrogation of protein functions, interactions and variant pathogenicity. However, current methodologies cannot efficiently engineer customizable sets diverse in endogenous loci across cellular contexts high throughput. Here, we combine cytosine adenine base editors a prime editor assess pathogenicity broad spectrum epithelial growth factor receptor gene (EGFR). Using pooled editing guide RNA libraries, install tens...
Cortical expansion in primate brains relies on enlargement of germinal zones during a prolonged developmental period. Although most mammals have two cortical zones, the ventricular zone (VZ) and subventricular (SVZ), gyrencephalic species display an additional zone, outer (oSVZ), which increases number diversity neurons generated corticogenesis. How oSVZ emerged evolution is poorly understood, but recent studies suggest role for non-coding RNAs, allow tight genetic program regulation...
Abstract Viral proteases and clinically safe inhibitors were employed to build integrated compact regulators of protein activity (iCROP) for post-translational regulation functional proteins by tunable proteolytic activity. In the absence inhibitor, co-localized/fused protease cleaves a target peptide sequence introduced in an exposed loop interest, irreversibly fragmenting structure destroying its functionality. We selected three demonstrated versatility iCROP framework validating it...
Significance We have long known that thyroid hormone (TH) stimulates formation of red blood cells and patients with diseases are often anemic, but the underlying molecular mechanisms unclear. This study uses pharmacologic genetic approaches in primary animal models to demonstrate essential roles nuclear receptor coactivator 4 (NCOA4) TH late erythropoiesis. show is for last steps culture, treatment drugs activate a particular receptor, TRβ, erythroid differentiation alleviates anemic...