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Molecular and Human Genetics

Houston, Texas

Department of Molecular and Human Genetics
Department of Molecular and Human Genetics
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Philip Ng, Ph.D.

Philip Ng, Ph.D.

Associate Professor of Molecular and Human Genetics

Other Positions

Associate Professor, Program in Translational Biology & Molecular Medicine


B.Sc., University of Toronto, 1986-1990
M.Sc., University of Guelph, 1991-1994
Ph.D., University of Guelph, 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. Brunetti-Pierri N, Ng T, Iannitti D, Cioffi W, Stapleton G, Law M, Breinholt J, Palmer D, Grove N, Rice K, Bauer C, Finegold M, Beaudet A, Mullins C, Ng P (2013). Transgene Expression up to 7 Years in Nonhuman Primates Following Hepatic Transduction with Helper-Dependent Adenoviral Vectors. Hum. Gene Ther. 24(8): 761-5. PubMed PMID: 23902403
  2. Brunetti-Pierri N, Liou A, Patel P, Palmer D, Grove N, Finegold M, Piccolo P, Donnachie E, Rice K, Beaudet A, Mullins C, Ng P (2012). Balloon catheter delivery of helper-dependent adenoviral vector results in sustained, therapeutic hFIX expression in rhesus macaques. Mol. Ther. 20(10): 1863-70. PubMed PMID: 22828499
  3. Brunetti-Pierri N, Ng P (2011). Helper-dependent adenoviral vectors for liver-directed gene therapy. Hum. Mol. Genet. 20(R1): R7-13. PubMed PMID: 21470977
  4. Dimmock D, Brunetti-Pierri N, Palmer DJ, Beaudet AL, Ng P (2011). Correction of hyperbilirubinemia in gunn rats using clinically relevant low doses of helper-dependent adenoviral vectors. Hum. Gene Ther. 22(4): 483-8. PubMed PMID: 20973621
  5. Vetrini F, Brunetti-Pierri N, Palmer DJ, Bertin T, Grove NC, Finegold MJ, Ng P (2010). Vasoactive Intestinal Peptide Increases Hepatic Transduction and Reduces Innate Immune Response Following Administration of Helper-dependent Ad. Mol. Ther. 18(7): 1339-45. PubMed PMID: 20461064
  6. Brunetti-Pierri N, Ng P (2010). Helper-dependent adenoviral vectors. In Parham, M and Bruinvels, J (Eds.) Gene Therapy for Autoimmune and Inflammatory Diseases. Birkhauser Publishing, Basel, Switzerland.
  7. Brunetti-Pierri N, Ng P (2009). Progress towards liver and lung-directed gene therapy with helper-dependent adenoviral vectors. Curr. Gene Ther. 9(5): 329-40. PubMed PMID: 19860648
  8. Brunetti-Pierri N, Ng P (2009). Helper-dependent adenoviral vectors for gene therapy. In Templeton, NS (Ed.) Gene Therapy: Therapeutic Mechanisms and Strategies, 3rd Edition. Marcel Dekker, Inc., New York, NY.
  9. Brunetti-Pierri N, Grove NC, Zuo Y, Edwards R, Palmer D, Cerullo V, Teruya J, Ng P (2009). Bioengineered Factor IX Molecules with Increased Catalytic Activity Improve the Therapeutic Index of Gene Therapy Vectors for Hemophilia B. Hum. Gene. Ther. 20(5): 479-85. PubMed PMID: 19196177
  10. Brunetti-Pierri N, Stapleton GE, Law M, Breinholt J, Palmer DJ, Zuo Y, Grove NC, Finegold MJ, Rice K, Beaudet AL, Mullins CE, Ng P (2009). Efficient, long-term hepatic gene transfer using clinically relevant HDAd doses by balloon occlusion catheter delivery in nonhuman primates. Mol. Ther. 17(2): 327-33. PubMed PMID: 19050700
  11. Palmer DJ, Ng P (2008). Methods for the production of first generation adenoviral vectors. Methods Mol. Biol. 433: 55-78. PubMed PMID: 18679617
  12. Palmer DJ, Ng P (2008). Methods for the production of helper-dependent adenoviral vectors. Methods Mol. Biol. 433: 33-53. PubMed PMID: 18679616
  13. Brunetti-Pierri N, Ng P (2008). Progress and prospects: gene therapy for genetic diseases with helper-dependent adenoviral vectors. Gene Ther. 15(8): 553-60. PubMed PMID: 18288209
  14. 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(4): 732-40. PubMed PMID: 17285138
  15. Flotte TR, Ng P, Dylla DE, McCray PB Jr, Wang G, Kolls JK, Hu J (2007). Viral vector-mediated and cell-based therapies for treatment of cystic fibrosis. Mol. Ther. 15(2): 229-41. (All authors contributed equally) PubMed PMID: 17235299
  16. Palmer DJ , Ng P (2007). Methods for the production and characterization of helper-dependent adenoviral vectors. In Friedmann T and Rossi J (Eds.) DNA Delivery/Gene Transfer Vector Lab Manual. Cold Spring Harbor Press.
  17. 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(5): 446-54. PubMed PMID: 17078387
  18. 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. 17(4): 391-404. (Journal cover) PubMed PMID: 16610927
  19. 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(7): 811-20. PubMed PMID: 16000063

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, MS BCM225
Houston, TX, 77030, U.S.A.

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

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