One potential treatment for CF is gene therapy, and a major
challenge in gene therapy is packaging replacement genes so they can be delivered to the target cells.
Not exact matches
«Today's approval marks another first
in the field of
gene therapy — both
in how the
therapy works and
in expanding the use of
gene therapy beyond the treatment of cancer to the treatment of vision loss — and this milestone reinforces the potential of this breakthrough approach
in treating a wide - range of
challenging diseases,» FDA Commissioner Scott Gottlieb said
in a statement.
«The
challenge has been how to direct certain
therapies designed to manipulate
genes of interest
in specific cells without developing a specific drug carrier for each specific cell type.
«Today's approval marks another first
in the field of
gene therapy — both
in how the
therapy works and
in expanding the use of
gene therapy beyond the treatment of cancer to the treatment of vision loss — and this milestone reinforces the potential of this breakthrough approach
in treating a wide - range of
challenging diseases,» FDA Commissioner Scott Gottlieb said
in a statement.
The symposium features presentations by Philippa Marrack and John Kappler talking on the T cell repertoire; William Paul on interleukin 4 as a prototypic immunoregulatory cytokine; Timothy Springer on lymphocyte trafficking; Pamela Bjorkman on structural studies of MHC and MHC - related proteins, and Jack Strominger on peptide presentation by class I and II MHC proteins; Thierry Boon on
genes coding for tumor rejection antigens, including the first tumor antigen, MAGE - 1; and Philip Greenberg on the modification of T cells for adoptive
therapy by retroviral - mediated
gene insertion Since then, the symposia series has attracted leading immunologists
in the cancer vaccine and antibody fields, providing them with a comprehensive view of the promises and
challenges in the development of cancer immunotherapies.
But efforts to develop adoptive T cell
therapies for solid tumors have hit upon a number of
challenges; the only
gene therapies to show significant benefit for patients have been
in liquid tumors — forms of leukemia and lymphoma.
Presentations included: Genetics Primer & Clinical Updates by Linford Williams, MS, LGC; Genetics and Women's Health: Seeing and Foreseeing the Ethical
Challenges Ahead by Ruth Farrell, MD, MA; Preimplantation Genetic Screening and Diagnosis: What You Need to Know by Marissa Coleridge, MS, LGC; Evolution of Prenatal Genetic Screening and Testing: NIPT and Beyond by Jeff Chapa, MD, MBA; Promises and Pitfalls of Prenatal Whole Exome Sequencing by Amanda Kalan, MD; Fertility Preservation and Cancer: Survivors, Previvors, and the Newly Diagnosed by Rebecca Flyckt, MD; Improving Access to Cancer Genetics via Telegenetics by Ryan Noss, MS, LGC; Breast Cancer: Management of Moderate Penetrance Predisposition
Genes by Holly Pederson, MD; Use of Hormonal and Non-hormonal
Therapies in Breast Cancer Survivors and Women at High Risk for Breast / Gyn Cancers by Holly Thacker, MD; Addressing Commonly Asked Patient Questions about Genetics by Rebekah Moore, MS, LGC, Christina Rigelsky, MS, LGC and Allison Schreiber, MS, LGC; and a panel discussion on Genetic Testing Reimbursement featuring Bruce Rogen, MD, MPH and John Yao, MD, MBA, MPH, which was moderated by Daniel Sullivan, MD..
In August, he will be the keynote speaker at the third Conference on Cell and
Gene Therapy for HIV cure at Fred Hutch about an even more
challenging goal: an HIV cure.