Целевая терапия и генная коррекция открывают новые перспективы лечения первичных иммунодефицитов

Autoimmune Disorders Awareness Exploring targeted therapy, other treatment options for primary immunodeficiencies Transcript Editor’s note: This is an automatically generated transcript. Please notify [email protected] if there are concerns regarding accuracy of the transcription. The vast majority of patients who have a primary immunodeficient disease really falls in the antibody compartment. These would be things like X-linked agammaglobulinemia, hyper-IgM syndrome, common variable immune deficiency. About 70% of all immune deficiencies occur in that antibody compartment, and the treatment for many of those antibody deficiencies is immunoglobulin replacement. So in immunology, we replace what's either missing or what doesn't work. About less than a third or so of patients have deficiency in the cellular compartment or combined cellular and antibody compartment, and then a minority have problems in the complement, compartment. So other, treatments may include antibiotic prophylaxis. This is where, you know, we give a low-dose antibiotic day after day after day to try and prevent an infection. Other types of treatment for more severe immune deficiencies include bone marrow transplant or hematopoietic stem cell transplant. Also, the evolution or advent of genetic diagnoses has opened up a new type of option for patients, and that's really targeted treatment. Targeted therapies are typically biologics or drugs that can be taken orally or by injection that once a genetic pathway is identified, these biologics are targeted therapies, and they either turn on or turn off the abnormal pathway and sort of restore the immune components and the immune function such that as long as the patient is getting treated with those medications, they can sustain long-term immune restoration and live really, you know, healthy and happy lives. Another, less common treatment for severe immune deficiencies is, gene therapy. Gene therapy has really gained some traction in the last five or ten years since some papers came out really showing a wide variety of successes in gene therapy. And so down the pipeline, you're likely to see more options for different genetic disorders using therapies like gene therapy.