Gene Editing for ALPK1 p.Thr237Met

ROSAH syndrome is a rare genetic disease caused by a mutation in the human alpha kinase 1 (ALPK1) gene (p.Thr237Met), leading to vision loss, swollen optic nerves, dry mouth, enlarged spleen, and frequent headaches. Researchers in the Laboratory of Clinical Immunology and Microbiology (LCIM) at the National Institute of Allergy and Infectious Diseases (NIAID) have developed a new approach that can precisely fix the ALPK1 mutation without causing unwanted changes in the patient’s DNA.

Treatment and Prevention of Inflammatory Bowel Disease (IBD) using Mutant and Chimeric IL-13 Molecules

Ulcerative colitis (UC) is a chronic inflammatory disease of the colorectum and affects approximately 400,000 people in the United States. The cause of UC is not known, although an abnormal immunological response to bacterial antigens in the gut microflora is thought to be involved. Present treatments for UC include anti-inflammatory therapy using aminosalicylates or corticosteroids, as well as immunomodulators and diet.

Muramyl Dipeptide as a Therapeutic Agent for Inflammation

The nucleotide-binding oligomerization domain 2 (NOD2) protein plays a key role in innate immunity as a sensor of muramyl dipeptide (MDP), a breakdown product of bacterial peptidoglycan. Bacterial peptidoglycan promotes the innate immune response through the activation of Toll-like receptor 2 (TLR2), which ultimately provokes inflammation. Activation of NOD2 by MDP negatively regulates the activity of TLR2, and thus reduces inflammation.

LRRK2 Inhibitors: Novel Treatment for Intestinal Bowel Disorders

Use of Leucine Rich Repeat Kinase 2 (LRRK2) inhibitors for the treatment of Intestinal Bowel Disorders (IBD) is disclosed. IBD is a broad term that describes conditions with chronic or recurring immune response and inflammation of the gastrointestinal tract. Crohn's disease and ulcerative colitis, two common forms of idiopathic IBD, are chronic, relapsing inflammatory disorders of the gastrointestinal tract.

Human Monoclonal Antibodies That Target Plasmodium Falciparum Sporozoites

Malaria is one of the worlds deadliest infectious diseases, causing an estimated 249 million cases and 608,000 deaths annually, with children in the regions of Africa and South Asia being most vulnerable. Approx 2,000 cases of malaria are reported in the United States each year, by travelers from malaria-risk countries. Malaria is a mosquito-borne parasitic disease transmitted through the bite of infected female mosquitoes, which introduces Plasmodium sporozoites into the bloodstream of the human host.

A Novel Strategy to Produce 6-cys Proteins Based on Pfs230D1 Domain Fusions

The Plasmodium parasite has a complex lifecycle during human infection and in the mosquito vector. Most advanced malaria vaccine candidates can confer only partial, short-term protection in malaria-endemic areas. A means of breaking the transmission of malaria to subsequent individuals could prevent a significant amount of human disease.

The primary embodiments of this technology are novel compositions of matter that produce enhanced transmission-blocking responses over current transmission blocking vaccines:

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