Seeking collaboration in oligonucleotide therapeutics (ASOs), neuromuscular diseases, and RNA biology

The Human Translational Genomics Group at INCLIVA Health Research Institute (Valencia, Spain) is interested in joining collaborative Horizon Europe consortia focused on rare diseases, neuromuscular disorders, RNA biology, precision medicine and ASO therapeutic approaches. The group is led by Ariadna Bargiela Schönbrunn.

Our research focuses on the molecular mechanisms underlying neuromuscular diseases, particularly myotonic dystrophy type 1 (DM1) and myotonic dystrophy type 2 (DM2), with strong expertise in RNA biology, alternative splicing regulation, miRNA-mediated pathogenic mechanisms and therapeutic development.

We have extensive experience in the identification and validation of therapeutic targets, development of cellular and animal disease models, biomarker discovery and translational research. A major focus of our work is the development and preclinical evaluation of antisense oligonucleotide (ASO)-based therapies. We actively coordinate and participate in national and international projects addressing oligonucleotide therapeutics and myotonic dystrophies.

Expertise offered to a consortium

• Design and preclinical development of antisense oligonucleotide (ASO) therapies.

• Neuromuscular and rare disease research.

• RNA biology and disease mechanisms.

• Alternative splicing analysis and spliceopathy research.

• miRNA biology and non-coding RNA-mediated regulation.

• Therapeutic target identification and validation.

• Myotonic dystrophy type 1 (DM1) and type 2 (DM2).

• Cellular and animal models for mechanistic and therapeutic studies.

• Biomarker discovery and translational genomics.

• Preclinical proof-of-concept studies for innovative therapeutic approaches.

• Multi-omics and systems biology.

  We are particularly interested in joining consortia where we can contribute to mechanistic studies, RNA biology work packages, target validation, biomarker development and preclinical evaluation of novel therapeutic strategies aimed at accelerating translation towards clinical applications.