Hemophilia Gene Therapy: Approaching the First Licensed Product
The clinical potential of hemophilia gene therapy has now been pursued for the past 30 years, and there is a realistic expectation that this goal will be achieved within the next couple of years with the licensing of a gene therapy product. While recent late phase clinical trials of hemophilia gene...
| Main Authors: | , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Wiley
2021-03-01
|
| Series: | HemaSphere |
| Online Access: | http://journals.lww.com/10.1097/HS9.0000000000000540 |
| _version_ | 1827337352218935296 |
|---|---|
| author | Paul Batty David Lillicrap |
| author_facet | Paul Batty David Lillicrap |
| author_sort | Paul Batty |
| collection | DOAJ |
| description | The clinical potential of hemophilia gene therapy has now been pursued for the past 30 years, and there is a realistic expectation that this goal will be achieved within the next couple of years with the licensing of a gene therapy product. While recent late phase clinical trials of hemophilia gene therapy have shown promising results, there remain a number of issues that require further attention with regard to both efficacy and safety of this therapeutic approach. In this review, we present information relating to the current status of the field and focus attention on the unanswered questions for hemophilia gene therapy and the future challenges that need to be overcome to enable the widespread application of this treatment paradigm. |
| first_indexed | 2024-03-07T18:51:41Z |
| format | Article |
| id | doaj.art-c9ba105b9a8543efb9e2034a960b5c2e |
| institution | Directory Open Access Journal |
| issn | 2572-9241 |
| language | English |
| last_indexed | 2024-03-07T18:51:41Z |
| publishDate | 2021-03-01 |
| publisher | Wiley |
| record_format | Article |
| series | HemaSphere |
| spelling | doaj.art-c9ba105b9a8543efb9e2034a960b5c2e2024-03-02T01:18:08ZengWileyHemaSphere2572-92412021-03-0153e54010.1097/HS9.0000000000000540202103000-00001Hemophilia Gene Therapy: Approaching the First Licensed ProductPaul Batty0David Lillicrap1Department of Pathology & Molecular Medicine, Richardson Laboratory, Queen’s University, Kingston, Ontario, Canada.Department of Pathology & Molecular Medicine, Richardson Laboratory, Queen’s University, Kingston, Ontario, Canada.The clinical potential of hemophilia gene therapy has now been pursued for the past 30 years, and there is a realistic expectation that this goal will be achieved within the next couple of years with the licensing of a gene therapy product. While recent late phase clinical trials of hemophilia gene therapy have shown promising results, there remain a number of issues that require further attention with regard to both efficacy and safety of this therapeutic approach. In this review, we present information relating to the current status of the field and focus attention on the unanswered questions for hemophilia gene therapy and the future challenges that need to be overcome to enable the widespread application of this treatment paradigm.http://journals.lww.com/10.1097/HS9.0000000000000540 |
| spellingShingle | Paul Batty David Lillicrap Hemophilia Gene Therapy: Approaching the First Licensed Product HemaSphere |
| title | Hemophilia Gene Therapy: Approaching the First Licensed Product |
| title_full | Hemophilia Gene Therapy: Approaching the First Licensed Product |
| title_fullStr | Hemophilia Gene Therapy: Approaching the First Licensed Product |
| title_full_unstemmed | Hemophilia Gene Therapy: Approaching the First Licensed Product |
| title_short | Hemophilia Gene Therapy: Approaching the First Licensed Product |
| title_sort | hemophilia gene therapy approaching the first licensed product |
| url | http://journals.lww.com/10.1097/HS9.0000000000000540 |
| work_keys_str_mv | AT paulbatty hemophiliagenetherapyapproachingthefirstlicensedproduct AT davidlillicrap hemophiliagenetherapyapproachingthefirstlicensedproduct |