Harnessing fibroblast heterogeneity for skin regeneration

Lentiviral vector process development and GMP manufacturing

Exploiting Adenovirus Biology for Advancing Adeno-Associated Viral Vector Manufacturing

Innovation Hubs for Gene Therapies initiative in the UK

Antisense approaches for the treatment of Duchenne muscular dystrophy and recent advances to address DMD brain comorbidities

Culture-acquired genetic changes in human pluripotent stem cells: implications for basic research and regenerative medicine

Gene therapy for an inherited childhood parkinsonism - dopamine transporter deficiency syndrome

Genetic therapies for retinal disease

A Self-regulating Gene Therapy for Rett Syndrome

Development of genetically refined “precision virotherapies” suitable for systemic anti-cancer applications

Gene therapy for rare surfactant protein deficiencies

Rescue of lethal SP-B deficiency in a murine model using lentiviral vector-mediated gene therapy

Generation of a de novo intronic junction in the DMD gene through CRISPR/Cas genome editing as a potential therapy for a high proportion of Duchenne Muscular Dystrophy patients.

Liver-directed lentiviral gene therapy is safe and curative in Argininosuccinic aciduria

Safety and efficacy analysis of in vivo lentiviral gene therapy for ARC syndrome

Traceless delivery of prime editor ribonucleoprotein (RNP) complexes in engineered nanoparticles

Developing a universal haematopoietic stem cell gene editing therapy for XIAP deficiency

Administration of mRNA alleviates disease biomarkers in a mouse model of pulmonary alveolar proteinosis (PAP)

Synthetic modified mRNA administration in a first in-human ex-situ heart failure model validates its use as a novel, effective and safe method of evaluating drug exposure and efficacy.

Molecular characterisation of a novel knock-out mouse model in the context of MPSIVA ex-vivo gene therapy development

Towards a scalable, closed and automated platform for the production of cost-efficient allogeneic cell therapies: showcase of an exemplar iNK process

IKC159V: A Next Generation Bicistronic Gene Therapy for the Treatment of Geographic Atrophy

Development of a precision immunovirotherapy expressing a folate receptor α bispecific immune cell engager for treatment of ovarian cancer

Single cell sequencing analysis of a CD28/CD40-based chimeric costimulatory antigen receptor (CoStAR™) activity reveals multiple functionally validated effector activities in CD4+ and CD8+ T cells.

Toward combined cell and gene therapy for airway epidermolysis bullosa

Non-viral GM-CSF Gene Therapy is an Effective Treatment in a Novel Murine Model of Autoimmune PAP

TROCEPT- an oncolytic virus platform engineered for αvβ6 integrin targeted tumour-localized expression of an immune checkpoint inhibitor following intravenous delivery.

Primary T-cell transduction performance as the guiding principle behind LV process development and platform innovation 

TROCEPT: a unique adenovirus for in-tumour expression of immunotherapies via intravenous delivery

αvβ6 Integrin Expression In Solid Tumours: A TROCEPT Target for Oncolytic Viral Therapy

Normothermically perfused human liver lobes containing tumours as a model to profile viral therapeutics.

Induced pluripotent stem cell derived alveolar type 2 cells as a model for testing novel gene therapies for interstitial lung diseases

Developing targeted extracellular vesicles for therapeutic delivery in cardiovascular disease.  

A bioluminescence-based lentiviral approach to identify regulators of the transcription factor TP63 in airway basal cells.

Compound screening in primary human airway basal cells identifies Wnt pathway modulators as potential pro-regenerative therapies

Surfactant air liquid interface model of ABCA3 surfactant protein deficiency

Commercial Scale Production (1000L) of AAV Using the Gibco™ CTS™ AAV-MAX Production System

Lenticlair™ 1: A Phase 1/2 trial evaluating the safety, tolerability and efficacy of an inhaled F/HN-pseudotyped lentiviral vector for CF gene therapy in people with CF ineligible for CFTR modulators 

Lentiviral gene therapy for fatal ABCA3 surfactant protein deficiency

Administration of lentiviral vector doses achieving high transduction efficiency is well tolerated in mice

Lenticlair™-ON: An extension trial examining long-term safety and efficacy outcomes associated with an inhaled F/HN-pseudotyped lentiviral vector for CF gene therapy in people with CF

Mitigating Indel Impact: Precision Genome Editing through Homology Independent Targeted Integration (HITI)

Functional analysis of gene-edited CF variant G542X

Cas9 nickase-mediated contraction of CAG/CTG repeats at multiple disease loci

Normothermically perfused livers as a model for profiling lentiviral pharmacokinetics and transduction

The new chromatin opening models as a strong potential tool for recombinant production and gene therapy applications

PKC agonists as small molecule inducing agents for enhancing lentiviral vector production

Targeted, non-viral delivery of gene editing technologies for the treatment of ASL deficiency

Conquering gene editing off-targets with Gibco™ CTS™ HiFi Cas9 Protein

Simple, rapid and robust bioluminescent cell-based assay for detecting neutralising antibodies against AAV in serum

Elevate Your CAR-T Therapy with Plasmid DNA Sanger Sequencing

CRISPR/Cas9-based cell engineering approaches to boost recombinant AVV productivity.

High throughput pegRNA screening platform using lentiviral delivery

AAV-Kir4.1 astrocyte gene therapy reduces seizure frequency in rodent models of acute and chronic epilepsy

AAV-shRNA mediated knockdown of CDKL5 to generate a novel human in vitro model of CDKL5 Deficiency Disorder

Synthetic, enzymatically produced DNA for gene therapy and vaccine applications

Transfer of an AAV production platform from bench-scale to 50 L: experiences, challenges and learnings.

Automation and comparability of qPCR and ddPCR for AAV genome titration for in-process samples.

Mechanistic and data driven models for optimisation and troubleshooting cell and gene therapy processes  

The TetraVecta™ System: A new tool kit enhancing lentiviral vector production for the next generation of gene therapies.

RevITTM A Universal AAV Enhancer and TransIT-VirusGEN® transfection reagent for Improved AAV Manufacturing.

High-throughput purification method for adeno-associated viruses using capture plates.

Integrated metrology approaches for the characterization of viral vectors: From mass spectrometry to molecular assays

A Novel, Perfusion-Based Lentiviral Vector Manufacturing Process Demonstrating Increased Productivity and Enhanced Purity

Comparison of droplet digital PCR and real-time quantitative PCR for lentiviral vector titration

Leveraging microfluidics for reproducible high lentiviral transduction efficiency

SpyVector; a modular capsid decoration platform to boost adenovirus vaccine-induced humoral immunity

Combined biolistic and cell penetrating delivery for an effective and scalable intradermal DNA vaccine 

Size-exclusion chromatography with multi angle light scattering (SEC-MALS) for AAV characterisation.

Bad and ugly? Mechanisms and implications for payload expression in lentivirus manufacture

Adeno-Associated Virus (AAV) Production: A Collaborative Approach to Accelerate Process Optimisation

Optimisation of a high throughput residual DNA Picogreen assay for analysing lysate samples from AAV production without GFP interference. 

Investigating the extracellular microenvironment as a potential target to monitor and control large-scale haematopoietic differentiation

Non-viral delivery of CRISPR activation tools for directed differentiation of induced pluripotent stem cells.

Developing stem cell and gene therapy for VPS33B deficiency

Development of a tumour selective precision immunovirotherapy expressing immune checkpoint inhibitors targeting LAG-3