Gene editing, personalized xenografts? Research horizons are expanding

September 8, 2015 | by Robin Heffler

Shared Resources The Chemical GMP Synthesis Facility at City of Hope is one of the institution's renowned shared resources. Soon, two new shared resources, known as cores, will debut.

City of Hope's shared resources are already the envy of many institutions ─ and that envy could soon grow.

Shared resources are offerings of specialized equipment, services and expert consultation that are referred to as "shared" because they're used by researchers from a variety of specialties. Such resources are the key to speeding up the discovery process, encouraging collaboration among researchers and exploring technologies.

Currently home to 26 shared resources, or cores, City of Hope is now launching two new specialized resources in response to requests from scientists and clinicians: the Gene Editing and Viral Vector Core and the Patient Derived Xenograft Core.

Together, they expand the abilities of City of Hope scientists to pursue even more research pathways for new treatments and cures for cancer, diabetes and other life-threatening diseases.

Engineering viruses to do good

The launch of the Gene Editing and Viral Vector Core is, in a way, a sign of victory in humankind's battle against viruses.

“Viruses have been tamed, and we now understand how to engineer them to manipulate genes in treating cancer,” said John Termini, Ph.D., City of Hope’s scientific director of shared resources.

“Researchers and clinicians can correct disease-causing genes by introducing new ones via gene therapy, or silence a cancer gene by editing it out,” he said. “Viral vectors are a very valuable research tool, and they’re becoming more valuable in patient treatment. For example, they have been used to successfully correct immune-system defects in patients with ‘Bubble Boy’ disease.”

The gene editing element of the core presents another approach to fighting cancer. Termini said that a new technology, called CRISPR-CAS9, enables relatively easy editing of genes in animal models with great accuracy.

An additional aspect of the core offers oncolytic pox viruses ─ viruses that are engineered to selectively infect and destroy cancer cells, while sparing nearby normal cells and tissues. This service has never been offered to the City of Hope research community. The core will be directed by renowned researchers with considerable expertise in these areas: Jiing-Kuan Yee, Ph.D., Saswati Chatterjee, Ph.D., and Nanhai Chen, Ph.D.

Creating a personalized approach to therapy

The Patient Derived Xenograft (PDX) Core is the second major new core.

Construction of a PDX involves removing a cancerous tumor from a specific patient, implanting it in mice with no immune system, and destroying the tumor by administering different drugs. The method offers a personalized approach to therapy by helping researchers to determine the most effective drugs for individual patients. It also overcomes the problem of some traditional drug studies that prove effective in animal models but not when tried on humans.

“PDX models are becoming increasingly popular in the cancer-research community,” Termini said. “It’s a way to preserve the characteristics of a person’s tumor in another model, and involves cooperation between surgeons, pathologists and veterinarians.”

At City of Hope, Termini noted, Steven T. Rosen, M.D., provost and chief scientific officer, supports the sharing of PDX models throughout the City of Hope research community. “This open research model is an excellent idea, will reduce duplication of efforts and, most important, will help validate the model and hopefully increase collaboration,” Termini said.

But wait. There's more ...

In the fall, City of Hope expects to debut a new and unique service  in the Integrative Genomics Core. Created by Pacific Biosciences, a new instrument, the RSII, will offer single-molecule real-time, or SMRT, technology for sequencing and analyzing DNA and RNA.

“With this, we will be the only comprehensive cancer center that will have ‘long-read’ technology,” Termini said. “Typical DNA sequencers can only read 100 letters at a time, and need extensive mathematical analysis to reassemble them so they can be interpreted. The new machine will be able to read up to 10,000 letters in a single run, with less reconstruction of the sequence information, which will enable researchers to discover new versions of cancer genes that were missed with the old technology.

“It will set City of Hope apart to have this tool, and more important, we will share this service among our fellow comprehensive cancer centers through our NCCN network. It will be an exciting opportunity for discovery and collaboration.”

Those two characteristics ─ discovery and collaboration ─ are what set City of Hope apart.

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Learn more about becoming a patient or getting a second opinion at City of Hope by visiting our website or by calling 800-826-HOPE (4673). You may also request a new patient appointment online. City of Hope staff will explain what's required for a consult at City of Hope and help you determine, before you come in, whether or not your insurance will pay for the appointment.

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