You work of a pharmaceutical company whose focus is new technologies and treatme

Question

You work of a pharmaceutical company whose focus is new technologies and treatments for autoimmune diseases. You first project led to a successful treatment for Scleroderma and now you are focused on drugs to treat IPEX. Acces the possibility of your team maunipulating the Scleroderma drug with minimal modifaction into a successful treatment for IPEX. Use your knowledge of Tregs in the immune response to list two symptoms that IPEX patients likely exhibit. Describe two possible strategies by which the presence of Tregs and/or the effect of Tregs may be achieved as a therapeutic to resolve IPEX symptoms.

Explanation / Answer

Answer:

IPEX - IPEX or immunodysregulation polyendocrinopathy enteropathy X -linked syndrome is a rare genetic disease linked to the dysfunction of the transcription factor FOXP3 (Forkhead box protein 3). It leads to the dysfunction of the regulatory T-cells (type of lymphocyte that plays a central role in cell-mediated immunity).

IPEX leads to autoimmune enteropathy, psoriasiform or eczematous dermatitis, autoimmune endocrinopathies, nail dystrophy, alopecia universalis and bullous pemphigoid.

Symptoms - Eczema, Hypothyroidism, Diarrhea, Lymphadenopathy, etc.

IPEX is actually an autoimmunity where body's own immune system attacks and plays against its own tissues and organs.

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