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01. Gene therapy Boulikas.pdf - Gene therapy & Molecular Biology

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<strong>Gene</strong> Therapy and <strong>Molecular</strong> <strong>Biology</strong> Vol 1, page 165<br />

Appendix 1. RAC-approved human gene <strong>therapy</strong><br />

protocols<br />

Using retrovirus, protocols: 1-117<br />

Using adenovirus: 118-157<br />

With naked plasmid: 158-164<br />

Adeno-Associated virus: 165, 166<br />

Immuno<strong>therapy</strong> or other: 167-186<br />

Cationic lipids: 187-220 on page 203-206 of following article (Martin and <strong>Boulikas</strong>, 1998)<br />

(Last updated: 21 November 1997)<br />

RETROVIRAL GENE<br />

DELIVERY<br />

Disease Protocol title Procedures Principal<br />

investigator<br />

1. <strong>Gene</strong> Marking /Cancer The Treatment of Patients with Advanced Cancer<br />

Using Cyclophosphamide, Interleukin-2 and Tumor<br />

Infiltrating Lymphocytes.<br />

2. <strong>Gene</strong> Therapy /Phase I<br />

/Monogenic Disease<br />

/Severe Combined<br />

Immune Deficiency due<br />

to Adenosine<br />

Deaminase Deficiency<br />

3. <strong>Gene</strong> Therapy /Phase I<br />

/Cancer /Melanoma<br />

/Immuno<strong>therapy</strong><br />

4. <strong>Gene</strong> Marking /Cancer<br />

/Acute Myelogenous<br />

Leukemia<br />

5. <strong>Gene</strong> Marking /Cancer<br />

/Neuroblastoma<br />

6. <strong>Gene</strong> Marking /Cancer<br />

/Neuroblastoma<br />

7. <strong>Gene</strong> Marking /Cancer<br />

/Chronic Myelogenous<br />

Leukemia<br />

8. <strong>Gene</strong> Marking /Acute<br />

Hepatic Failure<br />

9. <strong>Gene</strong> Marking /Cancer<br />

/Melanoma<br />

10. <strong>Gene</strong> Therapy /Phase I<br />

/Cancer /Melanoma<br />

Treatment of Severe Combined Immune Deficiency<br />

(SCID) due to Adenosine Deaminase (ADA)<br />

Deficiency with Autologous Lymphocytes Transduced<br />

with the Human ADA <strong>Gene</strong>: An Experimental Study.<br />

<strong>Gene</strong> Therapy of Patients with Advanced Cancer<br />

Using Tumor Infiltrating Lymphocytes Transduced<br />

with the <strong>Gene</strong> Coding for Tumor Necrosis Factor.<br />

Autologous Bone Marrow Transplant for Children<br />

with Acute Myelogenous Leukemia in First Complete<br />

Remission: Use of Marker <strong>Gene</strong>s to Investigate the<br />

<strong>Biology</strong> of Marrow Reconstitution and the Mechanism<br />

of Relapse.<br />

A Phase I /II Trial of High Dose Carboplatin and<br />

Etoposide with Autologous Marrow Support for<br />

Treatment of Stage D Neuroblastoma in First<br />

Remission: Use of Marker <strong>Gene</strong>s to Investigate the<br />

<strong>Biology</strong> of Marrow Reconstitution and the Mechanism<br />

of Relapse.<br />

A Phase II Trial of High-Dose Carboplatin and<br />

Etoposide with Autologous Marrow Support for<br />

Treatment of Relapse /Refractory Neuroblastoma<br />

Without Apparent Bone Marrow Involvement.<br />

Autologous Bone Marrow Transplantation for<br />

Chronic Myelogenous Leukemia in which Retroviral<br />

Markers are Used to Discriminate between Relapse<br />

which Arises from Systemic Disease Remaining after<br />

PreparativeTherapy Versus Relapse due to Residual<br />

Leukemic Cells in Autologous Marrow: A Pilot Trial.<br />

Hepatocellular Transplantation in Acute Hepatic<br />

Failure and Targeting <strong>Gene</strong>tic Markers to Hepatic<br />

Cells.<br />

The Administration of Interleukin-2 and Tumor<br />

Infiltrating Lymphocytes to Patients with Melanoma.<br />

Immunization of Cancer Patients Using Autologous<br />

Cancer Cells Modified by Insertion of the <strong>Gene</strong> for<br />

165<br />

In Vitro /Tumor Infiltrating Lymphocytes<br />

/Retrovirus /Neomycin Phosphotransferase cDNA<br />

/Intravenous<br />

In Vitro /Autologous Peripheral Blood Cells<br />

/CD34 + Autologous Peripheral Blood Cells /Cord<br />

Blood /Placenta Cells /Retrovirus /Adenosine<br />

Deaminase cDNA /Neomycin Phosphotransferase<br />

cDNA /Intravenous<br />

In Vitro /Tumor Infiltrating Lymphocytes<br />

/Retrovirus /Cytokine /Tumor Necrosis Factor<br />

cDNA /Neomycin Phosphotransferase cDNA<br />

/Intravenous<br />

In Vitro /Autologous Bone Marrow Cells<br />

/Retrovirus /Neomycin Phosphotransferase cDNA<br />

/Bone Marrow Transplant<br />

In Vitro /Autologous Bone Marrow Cells<br />

/Retrovirus /Neomycin Phosphotransferase cDNA<br />

/Bone Marrow Transplant<br />

In Vitro /Autologous Bone Marrow Cells<br />

/Retrovirus /Neomycin Phosphotransferase cDNA<br />

/Bone Marrow Transplant<br />

In Vitro /Autologous Bone Marrow Cells<br />

/Retrovirus /Neomycin Phosphotransferase cDNA<br />

/Bone Marrow Transplant<br />

In Vitro /Autologous Hepatocytes /Retrovirus<br />

/Neomycin Phosphotransferase cDNA<br />

/Intrahepatic<br />

In Vitro /Tumor Infiltrating Lymphocytes<br />

/Retrovirus /Neomycin Phosphotransferase cDNA<br />

/Intravenous<br />

In Vitro /Autologous Tumor Cells /Lethally<br />

Irradiated /Retrovirus /Cytokine /Tumor Necrosis<br />

Rosenberg,<br />

Steven A<br />

Blaese, R.<br />

Michael<br />

Rosenberg,<br />

Steven A<br />

Brenner,<br />

Malcolm K.<br />

Brenner,<br />

Malcolm K.<br />

Brenner,<br />

Malcolm K<br />

Deisseroth,<br />

Albert B<br />

Ledley, Fred D.<br />

Lotze, Michael<br />

T<br />

Rosenberg,<br />

Steven A

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