TS-000643 — Gene therapy experts at Nationwide Children’s Hospital have made significant advancements in designing optimal viral vectors for producing Good Manufacturing Practice (GMP)-grade viral vector products. Our experts have optimized properties of vectors for a wide variety of Adeno-associated virus (AAV) serotypes, including AAV1, 2, 2.5, 3, 5, 6, 8, 9 and rh74. In particular, our experts have optimized virus packaging efficiency, reduced potential to form replication competent AAV and replaced the beta-lactam resistant gene with kanamycin in order to be compliant with European Union (EU) regulations. Our experts have made additional optimized vectors for AAVrh74, and AAV9 that allow for more efficient purification and improved CNS transduction, respectively.

TS-000629 — Gene therapy experts at Nationwide Children’s Hospital have made significant advancements in designing optimal viral vectors for producing Good Manufacturing Practice (GMP)-grade viral vector products. Our experts have optimized properties of vectors for a wide variety of Adeno-associated virus (AAV) serotypes, including AAV1, 2, 2.5, 3, 5, 6, 8, 9 and rh74. In particular, our experts have optimized virus packaging efficiency, reduced potential to form replication competent AAV and replaced the beta-lactam resistant gene with kanamycin in order to be compliant with European Union (EU) regulations. Our experts have made additional optimized vectors for AAVrh74, and AAV9 that allow for more efficient purification and improved CNS transduction, respectively.

TS-000627 — Gene therapy experts at Nationwide Children’s Hospital have made significant advancements in designing optimal viral vectors for producing Good Manufacturing Practice (GMP)-grade viral vector products. Our experts have optimized properties of vectors for a wide variety of Adeno-associated virus (AAV) serotypes, including AAV1, 2, 2.5, 3, 5, 6, 8, 9 and rh74. In particular, our experts have optimized virus packaging efficiency, reduced potential to form replication competent AAV and replaced the beta-lactam resistant gene with kanamycin in order to be compliant with European Union (EU) regulations. Our experts have made additional optimized vectors for AAVrh74, and AAV9 that allow for more efficient purification and improved CNS transduction, respectively.

TS-000598 — CRIPSR/Cas9 gene editing technology is a promising tool for treating disease but requires the delivery of the large Cas9 enzyme. Gene therapy experts at Nationwide Children’s Hospital have taken two different approaches to couple the CRISPR gene editing machinery with AAV, including constructing Cas9 enzyme as a stable component of the AAV particle, as well as expressing Cas9 on the surface of the viral particle.

TS-000322 — CRIPSR/Cas9 gene editing technology is a promising tool for treating disease but requires the delivery of the large Cas9 enzyme. Gene therapy experts at Nationwide Children’s Hospital have taken two different approaches to couple the CRISPR gene editing machinery with AAV, including constructing Cas9 enzyme as a stable component of the AAV particle, as well as expressing Cas9 on the surface of the viral particle.

TS-000298 — Adeno-associated virus (AAV) vectors are replication defective viruses that are engineered to deliver therapeutic genetic cargo to cells. The structural and enzymatic AAV proteins are traditionally supplied “in trans” to generate engineered particles for gene delivery. One constraint of AAV vectors is the size limitation of the genetic insert. Gene therapy experts at Nationwide Children’s Hospital have engineered an AAV vector that expresses the Rep78 protein on the surface of the viral particle thus eliminating the need to package the rep coding region within the particle. This strategy allows for delivery of a functional Rep78 protein while increasing the overall therapeutic gene insert size.

TS-000288 — Gene therapy experts at Nationwide Children’s Hospital have made significant advancements in designing optimal viral vectors for producing Good Manufacturing Practice (GMP)-grade viral vector products. Our experts have optimized properties of vectors for a wide variety of Adeno-associated virus (AAV) serotypes, including AAV1, 2, 2.5, 3, 5, 6, 8, 9 and rh74. In particular, our experts have optimized virus packaging efficiency, reduced potential to form replication competent AAV and replaced the beta-lactam resistant gene with kanamycin in order to be compliant with European Union (EU) regulations. Our experts have made additional optimized vectors for AAVrh74, and AAV9 that allow for more efficient purification and improved CNS transduction, respectively.