Technologies

AIDD is an integrated tool and a radically new way to discovery new drugs for neurodegenerative diseases (Alzheimer’s, Epilepsy, Ageing, etc.).

The development of genome editing tools has revolutionized the way we think and deal with genetics. The use of Cas9 or its variants allows modifications of specific sites in the human genome by inducing deletions and insertions in a more or less controlled way. In recent years, a new class of tools for genome editing has emerged: the base editors (BE), which result from the fusion of a modified Cas9, which serves to direct the BE to the target, and an active deaminase acting on the DNA, which mediates the C> T or A> G editing.

Aptamers, short structured single-stranded oligonucleotides binding at high affinity to a given target protein, are selected from large combinatorial libraries through repeated cycles of incubation of the library with the target, recovery and amplification of target-bound oligonucleotides (SELEX technology, Systematic Evolution of Ligands by EXponential enrichment). SELEX can be applied to select aptamers against a known target protein or against a specific cell phenotype, without any prior knowledge of the specific target, leading to new biomarkers discovery.

The technology we participate to develop, called "Zinc-Finger Artificial Transcription Factors (ZF-ATFs)", allows to design, realize and select artificial genes coding for proteins capable of recognizing and binding "potentially" any DNA sequence. We used ZF-ATF technology to reprogram the expression of "beneficial" genes capable of efficiently counteracting the negative effect of mutated genes related to rare diseases.

Time-correlated single photon counting (TCSPC) is regarded as the “gold-standard” method for fluorescence lifetime measurements. However, TCSPC requires using highly sensitive detectors, not suitable for measurements under bright light conditions, thereby making the use impractical in clinical settings. The invention described here solves this problem by synchronizing the fluorescence detection with an external light source.

The Biocrystal Facility, a large multidisciplinary laboratory established at the Institute of Molecular Biology and Pathology (IBPM) of CNR, in collaboration with the Biochemistry Department of Sapienza University aims at supporting the italian scientists and the pharmaceutical companies in the research to find new drug and vaccine against the endemic and epidemic diseases through structure-based drug design.

The present invention relates to the biomedical sector of the treatment of lung diseases and related symptoms. In particular, the present invention provides compositions and methods based on the use of selected polymeric biomaterials, in combination with stem cells and/or their secretome, capable of synergistically improving the development, regeneration and repair of chronic lung injuries and related symptoms.

The virtual dynamic docking, carried out in the MOLBD3 lab of the Institute of Biophysics, allows the identification of new drugs through the structural information deriving from the study of target proteins, responsible for some human pathologies. In particular, we screen drugs or small molecules (commercial/own libraries) against known protein sites, surface cavities, surfaces of protein-protein interactions (fixed/rigid hotspots) or structural transition states (dynamic hotspots).

The herein described technology aims at the development of a platform of injectable hydrogels for application as drug carriers for localized delivery or in the regenerative medicine field. The use of ad-hoc synthesized poly(ether urethane)s (PEUs) as hydrogel forming materials is a common property which characterizes all the systems belonging to this platform.

In the last years, genetics played a strategic role in the identification of therapeutic targets for complex diseases. Genetic studies identified thousands of variants contributing to disease onset and/or to the influence of measurable features (phenotypes) impacting health. The mechanism of action by which they modulate diseases and phenotypes is still unknown for the vast majority.

Integrative omics has posed new challenges in modern precision medicine, particularly in oncology, including i) the identification of new tumor markers for early, precise, and non-invasive diagnostics, and ii) the discovery of innovative molecular targets for therapeutic applications. Our studies on medulloblastoma, a highly malignant childhood tumor, have contributed to identifying RNA molecules that meet these criteria.

The aim of the research group is the creation of 3D models (microorgan/ organoids) constructed using samples obtained from patients, both biopsy samples and samples collected with non-invasive techniques (exhaled breath condensate, induced sputum, blood samples).

An innovative approach for the treatment of diabetic and venous ulcers, characterized by a difficult healing process and therefore at potential risk of infection and therefore of hospitalization and amputation of the limb, is represented by the local administration of "bioactive" factors through the use of synthetic and/or biological matrices that allow a gradual and controlled release in order to obtain a better and faster healing.

Recently, nanoparticles and nanovesicles have been investigated as potential approaches for the treatment of neurodegenerative diseases. In particular, in the Biotech sector an increasingly deeper penetration of new treatment models and biological drugs based on cellular, subcellular and vesicle therapies is expected. The patent is based on the production of Myelin-based nanoVesicles (MyVes) produced by microfluidics, starting from myelin extracted from brain tissue. These vesicles find two major fields of applications as potential drugs or as supplements/nutraceuticals.

Severe asthma or chronic obstructive pulmonary disease (COPD) are nowadays associated with a poor response to corticosteroids which led to the use of high-dose with consequent improved onset of side effects. The use of nanotechnologies can represent an innovative approach for the effective treatment of both asthma and COPD. The development of new nano-formulations involving the use of nanomaterials and specifically tailored to be inhaled offers numerous advantages over conventional inhaled dosage forms.