Category Archives: Gene Therapy

Gene therapy is a novel approach to treat, cure, or ultimately prevent disease by changing the expression of a persons genes. Gene therapy is in its infancy, and current therapies are primarily experimental, with most human clinical trials still in the research stages. How does gene therapy work? Genes are composed of DNA that carries information needed to make proteins the building blocks of our bodies. Variations in the DNA sequence or code of a gene are called mutations, which often are harmless but sometimes can lead to serious disease. Gene therapy treats disease by repairing dysfunctional genes or by providing copies of missing genes. To reverse disease caused by genetic damage, researchers isolate normal DNA and package it into a vehicle known as a vector, which acts as a molecular delivery truck. Vectors composed of viral DNA sequences have been used successfully in human gene therapy trials. Doctors infect a target cell usually from a tissue affected by the illness, such as liver or lung cellswith the vector. The vector unloads its DNA cargo, which then begins producing the proper proteins and restores the cell to normal. Problems can arise if the DNA is inserted into the wrong place … Continue reading →

GenSight Biologics, a clinical stage biopharmaceutical company pioneering the development of gene therapy based treatments for retinal degenerative diseases, Pixium Vision (FR0011950641 - PIX), a company developing innovative Vision Restoration Systems (VRS) to allow patients who have lost their sight to lead more independent lives, and Fondation Voir et Entendre, a foundation for scientific cooperation aiming to boost the scientific and medical potential of the Institut de la Vision to meet the sensory challenges of hearing and vision impairments, join forces and announce that the SIGHT AGAIN project will receive a total of 18.5 million funding over five years as part of the Investment for the Future. SIGHT AGAIN, a collaborative research and development project, aims to restore vision to legally blind patients with retinitis pigmentosa at different stages. Retinitis pigmentosa is a rare and inherited disease, a group of genetic disorders that affect the retina and is characterized by progressive bilateral photoreceptor degeneration leading to blindness. Globally, more than 1.5 million people are affected by this irreversible disease, for which there is no cure to stabilize or restore vision. Coordinated by GenSight Biologics, SIGHT AGAIN aims to develop two complementary therapeutic products to restore vision: anoptogeneticgene therapy product and … Continue reading →

Drugmakers have a slew of treatments for afflictions related to sex and drugs. Now they may have one for rock n roll. Novartis AG (NOVN) is developing a gene therapy that may reverse hearing loss by stimulating the regrowth of microscopic hair cells in the inner ear, allowing people to hear. The hairs are destroyed by prolonged exposure to loud noise, and dont take root again naturally. Novartis treated the first patient in October after successful tests on rats. While hearing loss is most common in the elderly, rates are high in the music industry and the military, and rising among teenagers who listen to music at high volume. Almost 13 percent of children and adolescents under 19 in the U.S. have permanent damage caused by excessive exposure to noise, according to the Centers for Disease Control and Prevention. A little too much Lady Gaga, said Mark Fishman, the head of Novartis Institutes for BioMedical Research, which is developing the therapy. About 36 million people in the U.S. have some form of hearing loss, according to the Basel, Switzerland-based company. A solution could mean big money for Novartis and GenVec Inc. (GNVC), its partner in developing the drug. Global sales … Continue reading →

HOPE, HYPE REALITY: TERMS USED IN GENE THERAPY By: defeatHIV … Continue reading →

Published January 07, 2015 Novartis is diving deeper into the world of gene-based medicine by signing deals with two U.S. biotech companies, giving it access to a powerful new genome editing technology. The tie-ups with unlisted Intellia Therapeutics and Caribou Biosciences show the Swiss drugmaker's confidence in the potential of so-called CRISPR technology, both for making new medicines and as a research tool. CRISPR, which stands for clustered regularly interspaced short palindromic repeats, allows scientists to edit the genes of selected cells accurately and efficiently. It has created great excitement since emerging two years ago and is being tipped for a Nobel Prize. While current gene therapy approaches involve adding genes to affected cells, CRISPR opens up the possibility of correcting those cells' faulty genes in the lab before returning them to the patient. Translating that promise into new treatments will take many years but Novartis' decision to apply the technology in its research labs is an important endorsement, since the company is the world's largest drugmaker by sales. It is also a sign the Swiss group intends to be at the forefront of the nascent field, after recently establishing a new cell and gene therapies unit within the company. … Continue reading →

See Inside History explains why people with the malady, and their physicians, are cautious to believe that a cure is in sight HEATHER VAN UXEM LEWIS In 2011, a remarkable study in the New England Journal of Medicine detailed the successful treatment of six adults with haemophilia B, which is caused by a deficiency in the coagulation protein known as factor IX. All of the participants were able to eliminate or reduce the frequency of clotting-factor-replacement injectionsthe current standard treatment for the diseaseafter their livers began producing functional levels of factor IX. The experimental therapy came in the form of an adeno-associated virus (AAV) carrying a gene that encodes instructions for production of normal levels of human factor IX. Three trials of AAV-mediated gene transfer in patients with haemophilia B are ongoing, with high expectations. After more than 20 years of research on gene transfer, it is a promising time for haemophilia therapies. It now seems likely that a single-dose treatment for haemophilia B using an AAV or another gene-transfer technique will be a viable option for many people in the next decade or two. Yet haemophilia researchers are not inclined to speak enthusiastically of a cure. Part of that … Continue reading →

CHOP gene therapy expert fine-tunes protein signals, improves motor function and reduces brain shrinkage in a neurological disorder IMAGE:Beverly L. Davidson, Ph.D., a gene therapy expert, is the director of The Center for Cellular and Molecular Therapeutics at the Children's Hospital of Philadelphia. view more Credit: The Children's Hospital of Philadelphia By adjusting the levels of a key signaling protein, researchers improved motor function and brain abnormalities in experimental animals with a form of Huntington's disease, a severe neurodegenerative disorder. The new findings may lay the groundwork of a novel treatment for people with this fatal, progressive disease. "This research shows the intricate workings of a biological pathway crucial to the development of Huntington's disease, and is highly relevant to drug development," said study leader Beverly L. Davidson, Ph.D., director of The Center for Cellular and Molecular Therapeutics at The Children's Hospital of Philadelphia (CHOP). "Our results in animals open the door to a promising potential therapy, based on carefully manipulating the dysregulated pathway to treat this devastating human disease." She added that restoring proper balance to these delicate biological processes may offer even broader benefits in treating other neurological diseases, such as amyotrophic lateral sclerosis (ALS), fragile X mental … Continue reading →

B3 14 Genetic Engineering and Gene Therapy Foundation Tier Summary OCR Gateway Additional Science B3 Lesson 14 review on Genetic Engineering and Gene Therapy for the Foundation tier. By: Westlands Science … Continue reading →

B3 14 Genetic Engineering and Gene Therapy Higher Tier Summary OCR Gateway Additional Science B3 Lesson 14 review on Genetic Engineering and Gene Therapy for the Higher tier. By: Westlands Science … Continue reading →

Preliminary Results from Gene Therapy in Patients with Beta Thalassemia At the American Society of Hematology Annual Meeting, preliminary data using bluebird bio's gene therapy to treat beta thalassemia and sickle cell anemia was... By: Rare Disease Report … Continue reading →