A combination of nanotechnology and gene therapy yielded treatment to torpedo cancer in March, 2009. The School of Pharmacy in London is testing a treatment in mice, which delivers genes covered in nanoparticles to target and destroy hard-to-reach cancer cells.(Citation 23)(Citation 24)

Personalized Cancer Therapy

Gene Therapy can be used extensively to personalize medicine when being used itself as a treatment. A prime example of this may be cancer treatment. Joan Scott, deputy director of the Genetics & Public Policy Center in Washington DC remarks, "Oncology is absolutely farther down [the] road of personalized medicine than other areas". The fact that cancer is caused by uncontrollable cell growth in individual patients thus makes gene therapy a prime option as personalized medicine for cancer patients. Gene therapy allows scientists and researchers to pinpoint specific tumors on individual patients.

Because each patient has a unique genome, each patient receives a specialized treatment with gene therapy. Hospitals can amplify and read out single altered nucleotides - point mutations - in DNA sequences of tumor genes commonly implicated in certain cancer.

A key example of gene therapy potential can be seen in the case when researcher Marco Marra's group, at the British Columbia Cancer Agency, sequenced the entire genome of a tumor that had spread from one patient's mouth to his lung. The researchers found that the patient had mutations in a tumor-suppressor gene called PTEN, and also abnormally high expression in a gene downstream PTEN, called RET. This explained why the patient had not responded to the treatment with the drug erlotinib. This also fit with some earlier studies suggesting that patients with active PTEN responded better to ertonlib. Thus, instead of the traditional approach, Marra's team instead provided a recommendation for the patient to take sunitinib, which inhibits the protein made by RET. The patient's cancer significantly regressed.

Molecular pathologist John Lafrate best summarizes the unique potential of gene therapy to personalize medicine, saying "In the next few years, I think every major cancer center is going to work on this approach".(Citation 25)(Citation 26)

Curing Eye Disease

When crucial parts of the human experience, such as vision, enter the playing field with disorders, gene therapy can work with them as well. Recently, scientists made a striking discovery. Enzymes called Dicer enzymes cut double stranded RNA molecules into shorter pieces; they are crucial for gene-silencing pathways involving small RNAs. Examples of small RNAs include interfering RNAs or microRNAs - they are the most abundant classes of small RNAs in mammals. Without the use of Dicer enzymes, most of these RNAs cannot be synthesized. The researchers, who were interested in eye disease called age-related macular degeneration, found that patients with the condition have reduced levels of the Dicer enzyme present in their retinal pigment epithelium, or RPE. The RPE is an eye-specific tissue which is affected in geographic atrophy. Alu RNA sequences are increased in the eyes of patients with the condition. However, injecting viable cells into the eyes of mice led to more degeneration rather than expected treatment. Scientists then tried increasing levels of the Dicer enzyme and then inserting the Alu RNA sequences; Results then yielded no Alu RNA toxic effects. The research is significant for scientists all over the world working to find cures and treatments for vision restoration and regeneration.(Citation 27)

More recent treatments and research accomplishments include -

Gene Therapy for Metastatic Melanoma - This gene therapy can produce complete remission of metastatic melanoma in mice. Researchers at the Indiana University School of Medicine have used a modified lentivirus, a potent and viable viral vector, to introduce a potent anti-melanoma T cell receptor genes into the hematopoietic stem cells (bone marrow cells producing all blood and immune system cells) of mice. The T cell gene works to recognize a specific protein found on the surface of melanoma. It is isolated and cloned from a patient with melanoma. The genetically modified stems cells are then transplanted directly back into the hosts and found to eradicate metastatic melanoma for the entire life time of the mice.(Citataion 28)

Gene Therapy Treatment for Color Blindness - As reported in the journal Nature, researchers were able to successfully treat color blindness in monkeys, a treatment which holds immense potential for correcting this condition in human beings. In monkeys, full color vision requires two versions of the opsin gene carried on the X chromosome. One of the versions codes for a red-detecting photoreceptor. The other version codes for a green-detecting photoreceptor. As the male monkeys have only one X chromosome, they carry only one version of the gene and thus, they are inevitably red–green color blind. A similar type of deficiency accounts for most common form of dichromatic color blindness in humans. The researchers introduced human form of the red-detecting opsin gene into a viral vector, and injected the virus behind the retina of two male squirrel monkeys. After 20 weeks, the monkeys' color skills improved dramatically. Jay Neitz, of the University of Washington, Seattle, who was a member of the team which carried out the treatment, remarked, “Here is an animal that is a perfect model for the human condition.”(Citation 29)

Gene Therapy for Inherited Blindness - Researchers from UCL Institute of Ophthalmology and Moorfields Eye Hospital NIHR Biomedical Research Centre have demonstrated an experimental treatment which can improve sight for a type of inherited blindness.(Citation 30)

Gene Therapy + Nano-Particle Treatment for Cancer - Researchers from UCL Institute of Ophthalmology and Moorfields Eye Hospital NIHR Biomedical Research Centre have demonstrated an experimental treatment which can improve sight for a type of inherited blindness.