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This feature explores some of the therapies using adult stem cells, and looks at some of the experimental stem cell therapies that are going on today.

Fascinating narrative science that explores the next frontier in medicine and genetics through the very personal prism of the children and families gene therapy has touched. Eight-year-old Corey Haas was nearly blind from a hereditary disorder when his sight was restored through a delicate procedure that made medical history.  Like something from a science fiction novel, doctors carefully introduced viruses bearing healing genes into Corey's eyes-a few days later, Corey could see, his sight restored by gene therapy.

Researchers have for the first time used electrical pulses delivered from a cochlear implant to deliver gene therapy, thereby successfully regrowing auditory nerves. The research also heralds a possible new way of treating a range of neurological disorders, including Parkinson's disease, and psychiatric conditions such as depression through this novel way of delivering gene therapy.

Approximately half the patients have received all the 12 doses of gene therapy or placebo given at monthly intervals. The remaining will be completing their dosing over the next few months, with the final dose in the final patient due around June.

Like humans, some golden retrievers develop Duchenne muscular dystrophy (DMD), a hereditary muscle wasting condition that begins early in life. Using gene therapy, scientists were able to restore muscle function in dogs with the disease, according to a study published today (July 25) in Nature Communications.

"This modular case study tells the story of Dan and Annie, a married couple of Acadian ancestry who have a genetic form of deafblindness called Usher syndrome. They live in Southwest Louisiana, home of the largest population of DeafBlind citizens in the United States. Acadian Usher syndrome is caused by an allele of the USH1C gene that came to Louisiana with the first Acadian settlers from Canada who founded today's Cajun population. This allele's single nucleotide substitution creates an erroneous splice site that produces a defective cytoskeletal protein (harmonin) of the cochlear and vestibular hair cells and retinal photoreceptors. This splice site is the target of a promising gene therapy. The case study applies and connects Mendelian inheritance, chromosomes, cell division, vision and hearing, DNA sequences, gene expression, gene therapy and population genetics to a specific gene and its movement through generations of Dan and Annie's families.  After the introduction, each of the remaining sections can be used independently either for in-class team activities or out-of-class extensions or assignments over an entire year of introductory undergraduate biology. "

8:19 video The Nobel Prize in Physiology or Medicine 2015 goes to William Campbell & Satoshi Ōmura "for their discoveries concerning a novel therapy against infections caused by roundworm parasites" and Youyou Tu "for her discoveries concerning a novel therapy against Malaria".

2:27 video overview of gene therapy

Before the gene treatment, the animals experienced about five serious bleeding events a year. After receiving the novel gene therapy, though, they experienced substantially fewer bleeding events over three years, as reported in the journal Nature Communications.

Promising animal model work utilizing gene therapy.

Book Chapter of gene therapy, nice figure on different approaches.

A new study shows that gene editing using CRISPR/Cas9 technology can work in rhesus monkey embryos. The results, published in the current issue of Human Molecular Genetics, open the door for pursuing gene editing in nonhuman primates as models for new therapies, including pharmacological, gene-, and stem cell-based therapies, says Keith Latham, animal science professor at Michigan State University and lead author of the study.

GlaxoSmithKline has developed what looks like a cure for a rare and deadly disease, and it's going to cost $665,000 for a single dose. That sky-high price tag-more than twice the average price of a house-is likely to stir what's already a long-simmering debate in health care: How much should curative therapies cost, and how should society pay for them?

Five people living with HIV are currently free of the detectable virus after taking part in a new vaccine-based therapy.  The patients are also not taking daily antiretroviral drugs, with one having been drug free for a total of seven months; a milestone moment in a move towards a future where HIV treatment doesn't require ART.

start at 4:00 human genome project translation target rare diseases with gene therapy: 1)Leber's congenital amaurosis- blindness 2) congenitaal AADC deficiency- enzyme responsible for movement 3)  Muscular Dystrophy- Pompe Disease

The Path to Myozyme Beginning in the 1960s and fueled by the biotechnology revolution in 1980s, researchers at academic centers around the world initiated work to identify a treatment for Pompe - including therapies that could replace the missing GAA enzyme in patients. Based on these early efforts, from 1998-2002 Genzyme worked to advance promising research involving four different drug candidates to treat Pompe:

LUMIZYME® (alglucosidase alfa) is an enzyme replacement therapy for patients with Pompe disease (acid α-glucosidase (GAA) deficiency).

OpenBiome (full name Microbiome Health Research Institute Inc.) is a nonprofit organization dedicated to expanding safe access to fecal microbiota transplantation (FMT) therapies. Founded by a small team of microbiologists, public health advocates, and concerned citizens, OpenBiome aims to significantly reduce the practical barriers for clinicians providing FMTs, while connecting scientists across studies and disciplines. 

In the study, published Monday (May 1) in Nature Biotechnology,  Luo and colleagues set their sights on two fusions genes they had previously found to be associated with prostate cancer and various forms of rapid and invasive cancer, including liver tumors. Using a modified CRISPR-Cas9 tool that creates a single- rather than double-stranded break in DNA, they targeted the chromosomal breakpoints that form these fusion genes and replaced fusion DNA with a gene encoding the enzyme HSV1-tk. This enzyme effectively kills tumor cells by converting the drug ganciclovir into its active form, which then blocks DNA synthesis and leads to cell death. (Ganciclovir is used to treat cytomegalovirus in humans.)

This case study was developed for an introductory biology course with the goal of integrating content (specifically, structure/function, signaling pathways, and homeostasis) while reinforcing general critical thinking skills and the scientific method (generating hypotheses, evaluating evidence, and making predictions). The case is suitable for a flipped classroom and there are several videos associated with it. The case revolves around an obese two-and-a-half-year-old boy who won't stop eating. Students become familiar with some basic concepts related to obesity and leptin signaling through the videos that they watch before class. They then use class time to work through the case (delivered as an interactive slide show, including several clicker questions) to determine the genetic basis for this child's obesity and possible therapies to manage his weight. The case could also be adapted and expanded to be used in a physiology course to explore the interaction of various hormones that regulate appetite and metabolic rate or in a cell biology class to explore JAK-STAT signaling.