Pawing way to better healthcare through molecular genetics and fundamental research

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Angelman syndrome

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Pawing way to better healthcare through molecular genetics and fundamental research

Donate

Angelman syndrome

Vision

The human organism is a complex yet fragile system suffering from endogenous and exogenous diseases that take hundreds of thousands of lives every year and decrease the quality of life for millions of people. Cancer, genetic hereditary disorders, and bacterial and viral infections fuel research to discover, create, or adapt curative solutions. We aim to combine our experience in the fields of molecular genetics and cell biology to produce and offer solutions to treat human hereditary disorders such as Angelman syndrome, Snijders Blok–Campeau syndrome, and spinal muscular atrophy. Moreover, we aim to create a library of soil microorganisms (isolated from biotopes of Ukraine), serving as a source of natural compounds to treat infectious diseases and cancers, and as a source of biotechnological enzymes and Cas genes.

Our ambition

We stand to create a research unit gathering ambitious young scientists, who are established or learning specialists in field of molecular genetics, natural product discovery, and cell biology. We aim to elaborate on treatment solutions coming from nature (bacterial secondary metabolites) or engineered biosynthetic platforms – synthetic biology-derived microbial cell factories and eukaryotic cell factories (modified natural products and gene-therapy solutions). We are well educated, experienced, angry and voracious in our research ambitions.

Fields of interest

Angelman syndrome

A report describing AS was published in 1965 by a British pediatrician Harry Angelman [1] . AS is a genetic disorder causing neurodevelopmental disease with a conserved and recognizable...

Snijders Block-Campeau syndrome

SBCS is a recently discovered genetic neurodevelopmental disorder, first described in 2018 by clinical researchers Lot Snijders Blok and Philippe Campeau. Individuals with this syndrome typically present with ...

Spinal muscular atrophy

SMA is a genetic disorder characterized by weakness and wasting (atrophy) in muscles used for movement (skeletal muscles). It is caused by a loss of specialized nerve cells, called motor neurons, in the spinal...

Customized gene therapies for autism spectrum disorders caused by monogenic mutations

A report describing AS was published in 1965 by a British pediatrician Harry Angelman [1] . AS is a genetic disorder causing ...

Secondary metabolites from bacteria inhabiting extreme anthropogenic environments

SBCS is a recently discovered genetic neurodevelopmental disorder, first described in 2018 by clinical researchers Lot Snijders ...

Personalized medicine

Research directions

Assessment of SBCS distribution among the Ukrainian populations:

Creation of models for the experimental investigation of SBCS:

Development of
gene-therapeutic approaches for SBCS:

Foundation

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Our foundation is going to support the development of biomedical research focused on searching for gene-therapeutic solutions to threat and manage rare hereditary neurodevelopmental disorders, unravel the molecular mechanisms underlying these disorders, search for new antibiotic, anticancer, and other biologically active natural products by screening producing soil bacteria.

1) Create gene-therapeutic approaches to treat rare genetic disorders by expressing non-mutated alleles of corresponding genes, as well as by genome-editing using CRISPR-Cas9-mediated techniques.

2) Investigate molecular mechanisms of the etiology of rare neurodevelopmental genetic disorders, using cell and laboratory animal models.

3) Study the population genetics of rare neurodevelopmental disorders.

4) Isolate and characterize new soil bacteria, that belong to phyla Actinomycetota, Bacillota, and Myxococota in order to create a strain library, which would be screened for the production of novel natural compounds, biotechnological enzymes, and Cas-like genes.

5) Create gene-engineered platforms to produce known and modified natural products, including antibacterials, anticancer compounds, etc.

Importance

The last decade has been extremely challenging for fundamental and applied biological science in Ukraine. The annexation of Crimea and the military intervention by the russian federation in 2014, followed by the launch of a full-scale war in 2022, have led to stagnation and a lack of funding, curtailing the ambitions of Ukrainian young scientists working in the field of biology. The old infrastructure deteriorates and decays, while new projects go unfunded; talented scientists are forced to leave the field for better-paying jobs, and ineptitude and incompetence rise. As one aspect of our mission, we envision contributing to the renaissance of Ukrainian biomedical science by creating a novel hub in Lviv for the development of biomedical sciences.