Sjogren-Larsson Syndrome (SLS) Drug Discovery Service
Creative Biolabs is well-known in the field of neuroscience and has completed many innovative projects in this field. Now we can provide one-stop research services against Sjogren-Larsson syndrome (SLS) for our clients all over the world.
Introduction to Sjogren-Larsson Syndrome (SLS)
Sjogren Larsson syndrome (SLS) is a rare autosomal recessive disorder caused by mutations in the ALDH3A2 gene. The classic triad of ichthyosis, mental retardation, and spasticity is the predominant clinical feature. Mutations in the ALDH3A2 gene would result in a deficiency of fatty aldehyde dehydrogenase (FALDH), which presents important roles in the oxidation of medium and long-chain fatty aldehydes derived from fatty alcohols, phytanic acids, ether glycerolipids, as well as sphingolipids. Fatty aldehydes can covalently react with proteins and certain amino-containing lipids to produce an unusual aldehyde adduct, N-alkyl-PE (NAPE). In summary, the accumulation of long-chain fatty aldehydes and metabolism-related ether glycerolipids in the pathogenesis of SLS.
Fig.1 Magnetic resonance imaging of the brain in a 4year old child with Sjogren Larsson syndrome. (Bindu, 2020)
Animal Models of Sjogren-Larsson Syndrome (SLS)
Our established mouse models contain a spontaneous ALDH3A2 mutation, which causes a deficiency of fatty aldehyde dehydrogenase (FALDH). Modulation of JNK activation has been proven useful in animal models. These mouse models are invaluable for the study of pathogenesis and the testing of therapeutic approaches. Based on our mouse models, we also offer mechanism of action (MOA) studies for our clients.
Therapeutic Approaches of Sjogren-Larsson Syndrome (SLS)
Currently, there are no effective or curative treatments for patients with SLS. Existing strategies are mainly symptomatic and can only alleviate the symptoms.
- Symptomatic Treatment
The treatment methods for the cutaneous symptoms in SLS are always non-specific and mainly involve restoring the epidermal water barrier. Muscle relaxants, benzodiazepines, and anticholinergics are treatments for spasticity in patients with SLS.
- Prospective Therapeutic Options
Repair of specific metabolic defects associated with FALDH deficiency and correction of genetic defects by gene transfer is the most promising avenues for SLS therapy. There are a variety of potential therapeutic options including but not limited to:
- Fatty Aldehyde Scavengers
- PPAR Agonists
- ALDH Activators
- Antioxidants
- Retinoids and Vitamin D Analogues
- Modulation of Sphingosine Signaling Pathway
- Replacement of Cutaneous Lipids
- Reduction of Fatty Alcohol Production
- Dietary Therapy
- Inhibition of LTB4 Synthesis
- Gene Therapy
Services for Sjogren-Larsson Syndrome (SLS)
- In Vitro Services
- In Vivo Services
- Ex Vivo Services
- Discovery Services
- Development Services
Sjogren-Larsson Syndrome (SLS) Drug Discovery Service
Equipped with world-leading platforms and extensive experience, Creative Biolabs provides one-stop drug discovery service for our clients. From the basic target identification to the final IND-enabling, we can meet all the needs of customers.
Creative Biolabs is a leading service provider that focuses on neuroscience research. We can assist you in designing the best research outline customized to meet the requirements of clients' programs. If you are interested in our services and products, please do not hesitate to contact us for more details.
Reference
- Bindu, P.S. Sjogren-Larsson Syndrome: mechanisms and management. The Application of Clinical Genetics. 2020, 13: 13.
