V Noé

Universitat de Barcelona

Development of two new cationic liposomes TROPY and SPIDER for the transfection of therapeutic oligonucleotides in mammalian cells

H Bareke

Marmara University, Istanbul 

Targeting lung metastasis in triple negative breast cancer by CRISPR/Cas9 mediated Tenascin-C  silencing with a chitosan-based vector

Y Jiménez Martínez

Universidad de Granada

hokd as a potential tool for directed cancer gene therapy

O Quintana-Bustamante

CIEMAT; CIBERER; IIS-FJD, UAM 

Optimized PKLR-gene editing in human Hematopoietic Stem and Progenitor Cells to facilitate clinical gene editing therapy for Pyruvate Kinase Deficiency

G Guenechea

Division of Hematopoietic Innovative Therapies, Biomedical Innovation Unit, Centro de Investigaciones Energéticas Medioambientales y Tecnológicas (CIEMAT), Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER) and Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS-FJD, UAM), Madrid 28040. Spain.

Editing of human Dyskerin Pseudouridine Synthase 1 gene in hematopoietic stem cells: A new tool to address the treatment of bone marrow failure in patients with X-linked dyskeratosis congenita

I Ramos-Hernández

GENyO- Centro de Genomica e Investigacion Oncologica: Pfizer / Universidad de Granada / Junta de Andalucia; Fundacion Publica Andaluza Progreso y Salud

CX3CR1 intron 4 targeting provides a novel safe harbour for gene editing therapies strategies on haematopoietic progenitor and stem cells

B Perucha

GENyO- Centro de Genomica e Investigacion Oncologica: Pfizer / Universidad de Granada / Junta de Andalucia

Gene editing technologies for the improvement of the production of CD19-CAR-T cell-derived exosomes 

M C Casado-Rosas

Centro Nacional de Investigaciones Oncológicas

CRISPR/CAS9 approach targeting gene amplifications for efficient and selective elimination of cancer cells

M García

UC3M-CIBERER-CIEMAT; FJD

In vivo gene editing based on CRISPR/Cas9 adenoviral vector delivery in humanized mouse models of Recessive Dystrophic Epidermolysis Bullosa Bullosa skin

F Gómez-Garcia 

Health Research Institute of Santiago de Compostela; University of Santiago de Compostela

Prime Editing as a potential strategy for the treatment of Autosomal Dominant Polycystic Kidney Disease in murine models

S T Cervera Mayor

Instituto de Salud Carlos III; CIBERER

Therapeutic extracellular vesicles (TEVs) to delivery Cas9-sgRNA complex for Ewing sarcoma treatment

A Nieto-Sánchez

Centro Nacional de Investigaciones Oncológicas

Detection of Ewing sarcoma fusion oncogenes variants using CRISPR/Cas13