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In a first, scientists led by Kamel Khalili, director of the Comprehensive NeuroAIDS Center at Temple University, report in the journal Gene Therapy that they have for the first time successfully eliminated HIV genes from the genomes of mice and rats infected with the virus.

In the August 1 issue of CELL, researchers from the Gene and Stem Cell Therapy Program at Sydney's Centenary Institute revealed another function of introns, or noncoding nucleotide sequences, in DNA. They reported that gene-sequencing techniques and computer analysis allowed them to demonstrate how granulocytes use noncoding DNA to regulate the activity of a group of genes that determines the cells' shape and function.

This case study follows a young cystic fibrosis (CF) patient named Lucas. Through Lucas's story and interactions between his parents and pediatrician, students learn about the scientific background and basis of CF. By reviewing email correspondence between Lucas's parents and various doctors, students gain an overview of CF research. CF has become a model disease in certain undergraduate biology classrooms due to its relatively clear mechanism and genetic basis. This case asks students to come up with their own ideas to improve on an existing line of research - gene therapy - in treating CF. During the process, students will gain a better appreciation of the innovative nature of science and develop research skills such as finding, understanding and analyzing primary literature. The activity was originally designed for first- and second-year students as part of an extracurricular case competition, but may be used for any undergraduate biology level. The case assumes basic (high school level) knowledge of genetics, biochemistry, cell biology and physiology.

Blood vessel growth-or angiogenesis-is critical for developing fetuses and to heal wounds. But it is also an essential element of how cancerous tumors grow and spread. Here, see a step-by-step explanation of how the process works and how anti-angiogenesis therapies might stop tumors before they become deadly.

2:44 video using factory analogy for vision loss

LONDON, England- (24 July 1997)- Move over Dolly, and make room for Polly, the first sheep cloned by nuclear transfer technology bearing a human gene. Investors hope the lamb will make them a mint, but more importantly, the arrival of Polly could be good news for hemophiliacs and others who rely on expensive protein therapy of their diseases.

Overview of genetic engineering and business assessment of scientific feasibility.  Good overview of past gene therapy attempts.

Researchers have designed a nanoparticle-based therapy that is effective in treating mice with multiple myeloma, a cancer of immune cells in the bone marrow.

Millions of people suffer from asthma, a frightening disease that makes you feel like you're drowning. Using both mouse and human cells - and a humanized mouse to show how human type 2 innate lymphoid cells (ILC2) work in the body, researchers at Janssen Research and Development, Dana-Farber Cancer Institute and Harvard Medical School are figuring out ILC2's role in asthma. Their findings have important implications in the quest for effective asthma therapies!

WEDNESDAY, April 22, 2015 (HealthDay News) -- In a step toward personalized vaccines against cancer, scientists report they have developed an immune-system therapy that knocks out several types of aggressive tumors in mice. German researchers said the findings, reported April 22 in the journal Nature, could lead to a "blueprint" for developing tailored vaccines for a range of cancers. Such vaccines would be designed for individual patients, based on the specific genetic mutations in their tumors.

Research will make the study of diseases such as Parkinson's and Alzheimer's easier, and could lead to personalized therapies for a variety of neurodegenerative disorders. The nerve cells generated by this new method show the same functional characteristics as the mature cells found in the body, making them much better models for the study of age-related diseases such as Parkinson's and Alzheimer's, and for the testing of new drugs.

Nature's best kept secret is a wonder molecule called RNA. It is central to the origin of life, evolution, and the cellular machinery that keeps us alive. In this Lab you'll play the role of a molecular engineer by solving RNA folding puzzles. Then take your skills to Eterna, where you can design RNAs that could be at the heart of future life-saving therapies.

In this lesson, students explore some of the risks and benefits of gene-based medicine. They look at concerns related to genetic testing (which looks for particular genetic variations) and personal genome sequencing (which sequences the entire genome of an individual). Through videos and discussions, students learn about existing technologies for genetic testing and therapies. They also explore matters such as the emotional consequences of genetic testing, discrimination, and privacy issues. In small groups, students discuss scenarios and then share and analyze related opinions and concerns.

Scientists have genetically engineered monkeys so that they exhibit behaviors similar to autism, with a goal of testing potential therapies on the animals in hopes that their resemblance to humans will yield more answers about the disorder. The scientists found that the monkeys showed "very similar behaviors related to human autism patients, including repetitive behaviors, increased anxiety and, most importantly, defects in social interactions," said Zilong Qiu, a leader of the research at the Institute of Neuroscience at the Chinese Academy of Sciences in Shanghai. The team is now imaging the brains of the monkeys, he said, "trying to identify the deficiency in the brain circuits that is responsible for the autism-like behavior."

Similar to humans who have obsessive compulsive disorder, dogs exhibit compulsive behaviors, in which they might repetitively spin around, chase their tail, bark, chew, or suck on a toy or a part of their body for an excessive amount of time. In the latest study from Tufts University, researchers identified genetic pathways that increase the severity of canine compulsive disorders in Doberman pinschers. This discovery may lead to the development of improved treatments and therapies for obsessive compulsive disorder (OCD) in people. 

Research has revealed striking differences in the trillions of bacteria - a.k.a., the microbiome - in the intestines of children with and without autism. But the gut bacteria in individuals with autism aren't just different. Researchers at the California Institute of Technology have shown for the first time that they may actually contribute to the disorder. They reported in the journal Cell in December 2013 that an experimental probiotic therapy alleviated autism-like behaviors in mice and are already planning a clinical trial.

The worst cancer cells don't sit still. Instead they metastasize-migrate from their original sites and establish new tumors in other parts of the body. Once a cancer spreads, it is harder to eliminate. A study by developmental biologists offers a fresh clue to how cancer cells acquire the ability to invade other tissues-a prerequisite for metastasis. It reveals that invasion requires cells to stop dividing. Therefore, the two processes- invasion and proliferation-are mutually exclusive. The finding could inform cancer therapies, which typically target rapidly proliferating cancer cells.

In the 1950s, Idaho sheep ranchers had a problem. About a quarter of their lambs were being born dead or deformed-sometimes with a single eye centered in the middle of their forehead, like the Cyclops of Greek legend, or missing their upper jaw. There were times when the pregnant ewes couldn't give birth at all, carrying the mutated fetuses until they were surgically delivered or the ewes themselves died. Unbeknownst to the farmers, the cause was a poisonous plant, western false hellebore (Veratrum californicum). Eventually, the plant would inspire a successful cancer therapy-but at the time, the sheep were what mattered.

This interrupted case study for the flipped classroom applies evolutionary genetics research to human health. Students learn about a naturally occurring, but rare, allele of the CCR5 gene, CCR5-Δ32, which provides resistance to HIV. They use data from primary literature sources to predict and interpret worldwide patterns of CCR5-Δ32 frequency distribution. They then discuss how these allele frequency patterns may have been driven by selection imposed by various diseases or by other evolutionary mechanisms. Next, they test published data using Hardy-Weinberg equilibrium to examine if CCR5-Δ32 also provides genetic resistance to West Nile virus. Finally, they complete a jigsaw discussion of Nature News articles that report on how CCR5 research is being used to develop therapies to treat HIV. Originally written for the evolution portion of a yearlong biology series for undergraduate majors, the case is also appropriate for some non-majors biology courses or, with added complexity, upper-level evolution, genetics, or cell biology courses.

This flipped case study begins with a video in which a student reveals a family member's diagnosis with breast cancer and then considers the whirlwind of questions that arise in such a situation. Students are asked to relate to the main character and identify what questions they would have and what resources they could consult to seek information.  This sets the context for students to use websites and videos to investigate the nature of cancer, its causes, and progression. Students then learn about the major methods of cancer treatment (surgery, radiation therapy, and chemotherapy), how they work, and the limitations and side effects of each. The case concludes by addressing the claim of one of the characters that there is a conspiracy to hide a cure for cancer. The case would be implemented near the middle of a high school, non-majors, or introductory biology course after discussion of basic cell biology and the cell cycle.