Register Your Interest
GENOME EDITING
Engineering Life: Gene Editing from Research To The Clinic
The Gene Editing Stage examines challenges in achieving precise gene edits and advancing gene and cell therapies, with talks on mitigating off-target effects and overcoming safety concerns in clinical applications.
DAY 1
On Day 1, The Genome Editing Stage will focus on advances in genome editing technologies and translating genome editing into the clinic.
[topic] ADVANCES IN GENOME EDITING TECHNOLOGIES
-
Reproducibility & Reporting Guidelines for Genome Editing in Animal & Human Models
- How much do we know about the function of mammalian genes?
- Looking to the future of genome editing for animal models
- The LAGeR guidelines for Improving laboratory animal genetic reporting
- How it benefits and translate to human research
Speaker
Head of Unit, Genetic Engineering & Model Validation Department
Institut Clinique de la Souris
-
Omega Bio-tek: Removing the Barriers of Automating Plasmid DNA Extraction
- Introduction to Omega Bio-tek and Product Portfolio
- Plasmid DNA and Applications
- New Mag-Bind® Endo-free Plasmid Midi and Mini Kits
- Automation Capabilities
Sponsor
-
Design of CRISPR Experiments & Cell Line Engineering
- Implementation of CRISPR techniques including creation of cell lines with CRISPR deletions
- Considerations for CRISPR knock-in experiments
- Guidelines to choosing the best CRISP delivery for cell engineering
-
[break] Lunch
- Exhibition Floor: Wander around the vibrant exhibition floor to see the latest technologies and services on offer
- Food Options: Grab a sandwich at The Tree of Life Cafe or head on over to the Food Village for a range of hot food options, including Indian, Jamaican and Lebanese food
- Live Lounge Over Lunch: 15-minute technology-focused presentations
- Poster Zone: Browse the posters and reach out to potential new collaborators
- Talkaoke: The Flying Saucer of Chat, a fun and interactive pop-up show that offers you the opportunity to sit down with others and discuss any topic you like
[topic] ADVANCES IN GENOME EDITING TECHNOLOGIES
-
Removal of Antimicrobial Resistance Genes from Bacterial Strains and Communities Using CRISPR-Cas9
- Antimicrobial Resistance (AMR) is a major threat to human healthcare Often, AMR genes are carried on plasmids – bacterial mobile genetic elements.
- We engineer a broad host-range plasmid to encode CRISPR-Cas9 (pKJK5::csg). pKJK5::csg can be used to protect bacteria from uptake of AMR genes, or to resensitise them to antibiotics by removal of AMR plasmids.
- We further assess for which use-cases mobile CRISPR-Cas9 tools such as pKJK5::csg are most effective.
-
Mobile CRISPR-Cas9 tools are a novel tool in the arsenal to combat the mounting tide of AMR.
-
Quantifying Non-Specific Off-Target Activity By Base Editors
- Accurate editing quantification is essential for studying RNA editing, often limited by coverage and alignment problems.
- We discuss several common pitfalls and several strategies appropriate for coding and non-coding regions
-
Methods developed and lessons learned from the endogenous editome may facilitate better site-directed editing design.
-
Transposable Element Expression in Gene Expression during Early Mammalian Development
- How the genome controls transposable elements by epigenetic modifications in early development
- Using CELLO-seq to study locus-specific transposable element expression in single cells using long-read sequencing
- Functional tool such as CRISPRi to understand the role of locus-specific transposable element expression in development
-
[break] Break
- Speed Networking: Fun and useful sessions where you’ll rapidly meet other Festival attendees to immediately expand your network
- Food Options: Grab a coffee and a cake at The Tree of Life Cafe or head on over to the Food Village for a range of hot food options including Indian, Jamaican and Lebanese food
- Exhibition Floor: Wander around the vibrant exhibition floor to see the latest technologies and services on offer
- Talkaoke: The Flying Saucer of Chat, a fun and interactive pop-up show that offers you the opportunity to sit down with others and discuss any topic you like
[topic] GENOME EDITING TRANSLATION INTO THE CLINIC
-
Gene Therapy & Editing for Neurological Diseases
- Why we need gene therapy / gene editing for neurological diseases
- Current approaches available
- Innovative and novel genetic therapies for intractable neurological diseases using a variety of in vivo and ex vivo experimental techniques,
Speaker
-
Mapping the Functional Impact of Non-Coding Regulatory Elements in Primary T Cells through Single-cell CRISPR Screens
- “Primary T cell crisprQTL” is a scalable, single-cell functional genomics approach for mapping regulatory elements to genes in primary human T cells.
- This framework can facilitate the interrogation of immune disease GWAS hits.
-
The combination of experimental and QTL-based techniques is likely to address the variant-to-function problem.
-
Application & Clinical Translation of CRISPR/Cas9 Genetic Screens in Cancer Cells
- Cancer dependency maps to support target identification and drug discovery
- The discovery of Werner Syndrome helicase as a synthetic-lethal target in microsatellite unstable cancers
DAY 2
Day 2 on The Genome Editing Stage will focus on Personalised Cell & Gene Therapy.
[topic] PERSONALISED CELL & GENE THERAPY
-
A New Age of Genomic Therapies for Hearing Loss?
- Genetic diagnostics for hearing loss have become widely available in the UK, with better informed etiologies for hearing loss
- Several companies are pursuing gene therapies for hearing loss, and have reported success around the world for OTOF mutations
- There is likely to be a coming paradigm shift in how we manage genetic hearing losses in the near to medium term future
-
In Vivo Evaluation of Novel Synthetic Promoters for CNS Gene Therapy
- AAV gene therapy: a clinical reality
- Gene regulatory element engineering for targeted CNS delivery
- Evaluation of a novel dopaminergic neuronal promoter candidate
Speaker
Senior Research Fellow in the Genetic Therapy Accelerator Centre
UCL Queen Square Institute of Neurology
-
Gene Therapy for Lung Diseases
- Developing RNA-based therapeutics for airways disease
- Inhaled mRNA delivery using non-viral vectors
- Preclinical and clinical research related to advanced therapies for rare lung diseases, i.e., cystic fibrosis and anti-trypsin deficiency
-
[break] Lunch
- Exhibition Floor: Wander around the vibrant exhibition floor to see the latest technologies and services on offer
- Food Options: Grab a sandwich at The Tree of Life Cafe or head on over to the Food Village for a range of hot food options including Indian, Jamaican and Lebanese food
- Biodata Showcase: 15-minute technology-focused presentations
- Poster Zone: Browse the posters and reach out to potential new collaborators
- Talkaoke: The Flying Saucer of Chat, a fun and interactive pop-up show that offers you the opportunity to sit down with others and discuss any topic you like
[topic] PERSONALISED CELL & GENE THERAPY
-
Crack in the Creation, Prenatal Therapy for Congenital Blood Disorders
- Prenatal Therapy In utero Gene Therapy
- In utero Gene Therapy
- Prenatal Therapy for Sickle Cell Disease
Speaker
Clinical Senior Lecturer and Consultant in Maternal and Fetal Medicine
King's College London
-
Personalised Engineered Cell Therapies for Cystic Fibrosis
- Gene editing therapies for cystic fibrosis using gene editing, gene delivery, stem cell biology and cystic fibrosis epithelial biology
- Optimal methods for delivery of CRISPR/Cas9 formulations
-
Air-liquid interface models of the CF epithelium for identifying biological readouts and testing therapies
-
Cell Therapies for Cartlige Repair
- Cell-based therapies for the repair of cartilage defects ‘Omic (transcriptomic, quantitative proteomics and metabolomics) and bioinformatics approaches to identify better performing cells and for patient stratification
- Novel allogeneic therapies for the treatment of cartilage defects
- Cell-based therapies for the repair of cartilage defects
-
[break] Break
- Speed Networking: Fun and useful sessions where you’ll rapidly meet other Festival attendees to immediately expand your network
- Food Options: Grab a sandwich at The Tree of Life Cafe or head on over to the Food Village for a range of hot food options, including Indian, Jamaican and Lebanese food
- Exhibition Floor: Wander around the vibrant exhibition floor to see the latest technologies and services on offer
- Talkaoke: The Flying Saucer of Chat, a fun and interactive pop-up show that offers you the opportunity to sit down with others and discuss any topic you like
-
Stage Closed