Columbia University

Technology Ventures

A safe, retroviral packaging cell line for gene transfer in human cells

Technology #138

Retroviral vectors are an efficient method for the transfer of foreign genes into human cells. However, a major obstacle towards their use is the possibility that replication-competent viruses could form and proliferate, leading to harmful insertion and mutations in patient cells. By sequestering the genes necessary for intact retroviral production on two different plasmids, the packaging cell line described by this technology eliminates the possibility of such complications, creating a safe and efficient means of gene transfer. Importantly, the high efficiency of gene transfer observed with normal retroviral vectors is maintained. As many human diseases arise from inherited or sporadic gene mutations, gene transfer represents a potential therapeutic possibility to correct these mutations and alleviate their resulting diseases.

A two-plasmid retroviral packaging cell line enables safe and efficient gene transfer in human and animal cells.

Current methods for gene transfer in human cells rely on genetically engineered viruses which have the potential to form actual virus particles. If viral replication occurs, these newly formed virions may integrate at sites and activate potentially harmful genes (i.e. oncogenes). In order to avoid this possibility, additional mutations are made in the viral genome, often at the expense of efficiency. The two-plasmid retroviral packaging cell line described in this technology maintains a high efficiency of gene transfer comparable to native retroviruses, while simultaneously blocking virion production. This method provides the high efficiency and stringent safety required for gene transfer therapy, and for the genetic manipulation of isolated human cells of limited number, such as bone marrow stem cells.

Lead Inventor:

Arthur Bank, M.D.

Applications:

  • Method for gene transfer in human and animal cells.
  • Safely package genetic material by preventing viral production and replication. *Therapeutic tool for the treatment of a wide variety of human diseases whose etiologies are based on genetic mutation.
  • Method of generating transgenic human/animal cell lines and animal models.

Advantages:

  • Distinct dual retroviral packaging ensures safety against undesired viral replication
  • Maintains high gene transfer efficiency comparable to native retroviruses

Patent information:

Patent Published(WO/1989/007150)

Licensing Status:

Available for licensing and sponsored research support

Tech Ventures Reference: IR 138

Related Publications:

Further Information:
Columbia | Technology Ventures Email: TechTransfer@columbia.edu