Assistant Professor, Department of Molecular and Human Genetics

B.Sc., University of Toronto, Canada, 1986-1990
M.Sc., University of Guelph, Canada, 1991-1994
Ph.D., University of Guelph, Canada, 1994-1999
Postdoctoral fellow, McMaster University, 1998-2001

RESEARCH INTERESTS:

My laboratory is interested in developing gene therapies for genetic diseases using helper-dependent adenoviral vectors (HDAd). HDAd (also called gutless or gutted adenovirus) do not contain any viral genes and thus represent a major improvement over early generation adenoviral vectors with respect to safety and efficacy. These vectors can transduce target cells with high efficiency to provide high level long-term transgene expression without chronic toxicity. Studies into improving the production of HDAd as well as their characterization are ongoing in my laboratory including manufacturing the vector under current Good Manufacturing Practices (cGMP) for clinical applications in humans.

A major focus of my laboratory is liver-directed gene therapy using HDAd to treat a wide variety of genetic disease such as hemophilia, Crigler-Najjar syndrome, cardiovascular disease, alpha 1-antitrypsin deficiency and many others. We are investigating novel methods of delivering HDAd preferentially into the liver of mice, dogs and nonhuman primates. Recently, we have developed a minimally invasive, balloon occlusion catheter-based method to deliver HDAd preferentially into the liver of large animals which results in negligible toxicity and long-term, high level transgene expression. We believe that this technology will pave the way towards human clinical application for a wide variety of genetic and acquired diseases. In collaboration with Dr. Arthur Beaudet, we are currently in the process of obtaining regulatory approval with the US Food and Drug Administration (FDA) to implement this novel method of delivering HDAd to treat hemophilia patients as well as possibly patients with Crigler-Najjar syndrome Type I. We are also investigating ways of modifying the capsid of the vector to achieve preferential transduction of hepatocytes.

Another major focus of my laboratory is lung-directed gene therapy using HDAd with the primary goal of treating cystic fibrosis. In collaboration with Dr. Peter Hiatt, a pediatric pulmonologist, we have recently developed a novel method of aerosolizing HDAd into the lungs of nonhuman primates which has resulted in very high efficiency gene transfer to the airway epithelium with negligible toxicity. Based on these encouraging and compelling results we plan to obtain regulatory approval from the FDA to use this novel technology to treat patients with cystic fibrosis in the near future.

We are also interested in investigating the innate and adaptive immune responses to HDAd. These important studies will provide information regarding the host-vector interactions which will be very useful for further improving the safety and efficacy of HDAd-mediated gene therapy.

SELECTED PUBLICATIONS:

1. Palmer DJ, Ng P (2008). Methods for the production of first generation adenoviral vectors. Methods Mol. Biol. 433: 55-78.

2. Palmer DJ, Ng P (2008). Methods for the production of helper-dependent adenoviral vectors. Methods Mol. Biol. 433: 33-53.

3. Brunetti-Pierri N, Ng P (2008). Progress and prospects: gene therapy for genetic diseases with helper-dependent adenoviral vectors. Gene Ther. 15: 553-560.

4. Brunetti-Pierri N, Stapleton GE, Palmer DJ, Zuo Y, Mane VP, Finegold MJ, Beaudet AL, Leland MM, Mullins CE, Ng P (2007). Pseudo-hydrodynamic delivery of helper-dependent adenoviral vectors into nonhuman primates for liver-directed gene therapy. Mol. Ther. 15: 732-740.

5. Brunetti-Pierri N, Ng P (2006). Progress towards the clinical application of helper-dependent adenoviral vectors for liver and lung gene therapy. Curr. Opin. Mol. Ther. 8: 446-454.

6. Brunetti-Pierri N, Ng T, Iannitii DA, Palmer DJ, Beaudet AL, Finegold MJ, Carey KD, Cioffi WG, Ng P (2006). Improved hepatic transduction, reduced systemic vector dissemination and long-term transgene expression by delivering helper-dependent adenoviral vectors into the surgically isolated liver of nonhuman primates. Hum. Gene Ther. 15: 35-46 (Journal Cover).

7. Brunetti-Pierri N, Nichols TC, McCorquodale S, Merricks E, Palmer DJ, Beaudet AL, Ng P (2005). Sustained phenotypic correction of canine hemophilia B following systemic administration of helper dependent adenoviral vectors. Hum. Gene Ther. 16: 811-820.

8. Brunetti-Pierri N, Palmer DJ, Mane V, Finegold M, Beaudet AL, Ng P (2005). Increased hepatic transduction with reduced systemic dissemination and proinflammatory cytokines following hydrodynamic injection of helper-dependent adenoviral vectors. Mol. Ther. 12: 99-106 (Journal Cover).

9. Palmer DJ, Ng P (2005). Helper-dependent adenoviral vectors. Hum. Gene Ther.16: 1-16.

10. Palmer DJ, Ng P (2004). Physical and infectious titers of helper-dependent adenoviral vectors: A method of direct comparison to the adenovirus reference material. Mol. Ther. 10: 792-798.

11. Brunetti-Pierri N, Palmer DJ, Beaudet AL, Carey D, Finegold M, Ng P (2004). Acute toxicity following high-dose systemic injection of helper-dependent adenoviral vectors into non human primates. Hum. Gene Ther. 15: 35-46 (Journal Cover).

12. Palmer DJ, Ng P (2003). Improved system for helper-dependent adenovirus vector production. Mol. Ther. 8: 846-852.

For more publications, see listing on Pub Med.

AWARDS AND HONORS:

2007: American Society of Gene Therapy Outstanding New Investigator Award

CONTACT INFORMATION:

Philip Ng, Ph.D.
Department of Molecular and Human Genetics
Baylor College of Medicine
One Baylor Plaza, Rm. T619
Houston, Texas 77030, U.S.A.
Mail Stop: BCM225

Phone: 713-798-4158
Fax: 713-798-7773
E-mail:

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