Gene Therapies

Jan 24, 2025

The 43rd Annual J.P. Morgan Healthcare Conference in 2025 showcased the accelerating progress of cell and gene therapies, reaffirming their transformative potential in modern medicine. Despite prior challenges, the field is now demonstrating tangible success, heralding a new era in treating complex and previously u...

Oct 23, 2024

Plasma protein therapeutics are revolutionizing the landscape of modern medicine by harnessing the healing potential of the human body. Derived from donated blood plasma, these life-saving treatments are packed with antibodies, clotting factors, and other essential proteins that help manage a wide array of conditio...

Aug 23, 2024

Neuromuscular disorders (NMD) encompass a broad spectrum of conditions impacting the peripheral nervous system, which comprises all motor and sensory nerves linking the brain and spinal cord to the body. These disorders comprise various classes of diseases, such as muscular dystrophies, myopathies, motor neuron dis...

Mar 04, 2024

Gene therapy is becoming a promising solution for retinal degenerative illnesses, particularly because the retina offers an excellent avenue for studying and treating eye-related issues. Importantly, it is the first tissue in the United States to receive approval for gene therapy in cases of inherited disorders. ...

Feb 05, 2024

Gene therapy is an innovative medical approach that manipulates an individual’s genetic material to prevent or treat diseases. It primarily involves introducing, modifying, or substituting genes within a patient’s cells. The central objective of gene therapy is to rectify genetic abnormalities, insert missing genes...

Oct 27, 2023

Hemophilia B is a rare genetic bleeding disorder in which affected individuals have insufficient levels of a blood protein called factor IX. Around 3 in 100 individuals with hemophilia B produce an antibody to the factor IX replacement therapy used to treat or avoid their bleeding episodes, called an inhibitor. The...

Jun 16, 2023

Dystrophic epidermolysis bullosa (DEB) is an inherited disorder caused by changes in the COL7A1 gene. In this rare condition, blisters form on the skin and the moist inner lining of some organs and body cavities. Depending upon the nature of the inheritance pattern, dystrophic epidermolysis bullosa is divided into ...

Nov 18, 2022

Gene therapy is an experimental technique that introduces functional genes into a patient’s body to counteract or replace defective ones, thereby curing disease without using pharmaceuticals, radiotherapy, or surgery. Genetic defects that are difficult to treat with drugs or antibodies can be treated with therapy w...

Apr 15, 2019

Osteoporosis is a bone disease that makes bones weak and brittle. Osteoporosis, as the word says itself “porous bones”. As we age, bone density decreases and even mild stress or a light fall can cause severe damage. If this happens excessively, the result is Osteoporosis. The older bones keep on degrading, and new ...

Apr 12, 2019

Gene Therapy is a way to introduce a normal working gene in the place of a mutated one which might result in some kinds of abnormalities. Gene Therapy is a novel way to express novel proteins, and re-design the genome to prevent any disease. The concept here is some disorders are caused due to their defect in a sin...