New core programs accelerate pace of research

The Harvard Stem Cell Institute brings together more than 750 scientists who share a common goal—to realize the promise of stem cell research to find novel treatments for some of humankind’s most devastating diseases.

To achieve this goal as rapidly and efficiently as possible, HSCI provides funding for core facilities that provide its entire research community with shared access to critical state-of-the-art technologies, expertise, and services that are beyond the means of any individual laboratory or institution.

During its first few years, HSCI funded core facilities for flow cytometry and human embryonic stem cells, both of which are vital to many stem cell scientists’ research. Last year, HSCI made major investments in two new, large-scale core programs that are now available to the HSCI community: the Therapeutic Screening Center and the Genome Modification Facility.

Therapeutic Screening Center: From Bench to Bedside

The Therapeutic Screening Center (TSC), led by Lee Rubin, PhD, starts by providing specialized expertise and technologies needed to identify the factors that direct adult and embryonic stem cells to proliferate and differentiate. This knowledge is the first step in the process of identifying or developing drugs to be used directly in patients to stimulate or inhibit proliferation of their own stem cells, or to direct stem cell differentiation in the laboratory to produce cells for transplantation.

Currently, the TSC is focusing on several neurological disorders, including a childhood motor neuron disease called spinal muscular atrophy, and is making “real progress” in this area, says Rubin. In the future, work in the TSC has the potential to lead to many other advances, such as the discovery of drugs that stimulate the proliferation of hematopoietic (blood-forming) stem cells for cancer patients undergoing bone-marrow transplantation, or that direct the differentiation of embryonic stem cells into insulin-producing cells for people with diabetes.

In addition, the TSC plans to use diseasespecific stem cells created by somatic cell nuclear transfer to identify agents that could halt or slow the progression of specific diseases; this work is also beginning with a focus on select neural diseases.

According to Rubin, in an era when biotechnology firms are reluctant to invest in new or risky projects, the TSC is ideally suited to help speed the translation of stem cell research from bench to bedside for the benefit of patients.

Genome Modification Facility: Making Mice

Headed by Manfred Baetscher, PhD, the Genome Modification Facility (GMF) uses highly sophisticated techniques and equipment to create novel strains of mice (transgenic mice) that are vital to stem cell research.

Transgenic mice are a critical tool to help scientists readily identify, isolate, manipulate, and genetically modify stem cells for their research. But the often-delicate procedures used to create these mice require technical skills, expensive micromanipulation equipment, and specialized procedures that are beyond the capabilities of most laboratories.

The GMF also creates mouse models of specific diseases, allowing researchers to evaluate the ability of stem cells to participate in the cell-based repair of those conditions.

According to HSCI faculty member Andrew McMahon, PhD, who oversees the facility, in addition to meeting the demand for genetically modified mice in a timely manner, the GMF will provide other valuable services to HSCI researchers and develop new approaches to genetic analysis.