Human Gene Therapy Clinical Development

Aims & Scope

Human Gene Therapy (HGT) is the premier, multidisciplinary journal covering all aspects of gene therapy.

The Journal publishes important advances in DNA, RNA, cell and immune therapies, validating the latest advances in research and new technologies.

Abstracting & Indexing

Journal is indexed in leading academic databases, ensuring global visibility and accessibility of our peer-reviewed research.

• Scopus
• Google Scholar

Subjects & Keywords

Journal’s research areas, covering key disciplines and specialized sub-topics in Medicine, designed to support cutting-edge academic discovery.

Most Cited Articles

The Most Cited Articles section features the journal's most impactful research, based on citation counts. These articles have been referenced frequently by other researchers, indicating their significant contribution to their respective fields.

• Lessons Learned from the Clinical Development and Market Authorization of Glybera

  Citation: 156

  Authors: Laura M., Devin M., April R., Christian, Jesse L., Jenessa B., Elizabeth A., Josh, Adam P., James M.

• AAVrh.10-Mediated APOE2 Central Nervous System Gene Therapy for APOE4-Associated Alzheimer's Disease

  Citation: 135

  Authors: Jonathan B., Michael G., Bishnu P., Alvin, Thomas, Christiana, Eduard, Lingzhi, Rodolfo J., Sebastien, Jonathan P., Douglas J., Stephen M., Dolan, Gregory A., Steven M., Ronald G.

• Design of a Phase I Clinical Trial to Evaluate M032, a Genetically Engineered HSV-1 Expressing IL-12, in Patients with Recurrent/Progressive Glioblastoma Multiforme, Anaplastic Astrocytoma, or Gliosarcoma

  Citation: 129

  Authors: Daxa M., Paul M., L. Burt, Kristen O., G. Yancey, James M.

• Persistent Expression of Dopamine-Synthesizing Enzymes 15 Years After Gene Transfer in a Primate Model of Parkinson's Disease

  Citation: 126

  Authors: Yoshihide, Ken-ichi, Kunihiko, Yuko, Fumiko, Naomi, Mika, Keiya, Shin-ichi

• Long-Term Follow-Up of a Phase I/II Study of ProSavin, a Lentiviral Vector Gene Therapy for Parkinson's Disease

  Citation: 124

  Authors: Stéphane, Jean Marc, Hélène, Katy, G. Scott, Sarah, Gaëtane, Philippe, Philip C., Philippe, Nicola J., Roger A., Kyriacos A.

• Design of a Phase I Clinical Trial to Evaluate Intratumoral Delivery of ErbB-Targeted Chimeric Antigen Receptor T-Cells in Locally Advanced or Recurrent Head and Neck Cancer

  Citation: 108

  Authors: May C.I., Sophie E., Jean-Pierre, Teresa Guerrero, James F., John

• Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-<i>hGAA</i>) Gene Therapy in Children Affected by Pompe Disease

  Citation: 96

  Authors: Manuela, Cristina, Barbara K., Lee Ann, Ibrahim S., Thomas J., Kirsten E., Saleem, Roland W., David D., Shelley W., Barry J.

• A Multicenter, Double-Blind, Phase III Clinical Trial to Evaluate the Efficacy and Safety of a Cell and Gene Therapy in Knee Osteoarthritis Patients

  Citation: 93

  Authors: Myung-Ku, Chul-Won, Yong, Sung-Do, Eui-Sung, Jeong-Ku, Ju-Hong, Jae-Doo, Seong-Il, Choong-Hyeok, Hee-Soo, Myung-Chul

• Intracerebral Gene Therapy Using AAVrh.10-hARSA Recombinant Vector to Treat Patients with Early-Onset Forms of Metachromatic Leukodystrophy: Preclinical Feasibility and Safety Assessments in Nonhuman Primates

  Citation: 84

  Authors: Michel, Françoise, Marie-Anne, Sylvie, Jack-Yves, Johan, Benoit, Frédérique, Ivan, Ingrid, Dolan, Mark M., Nathalie, Philippe, Ronald G., Thomas, Caroline, Patrick

• Evaluation of Readministration of a Recombinant Adeno-Associated Virus Vector Expressing Acid Alpha-Glucosidase in Pompe Disease: Preclinical to Clinical Planning

  Citation: 82

  Authors: Manuela, Brian, Nathalie, Thomas J., Kaitlyn J., Gensheng, Janet, Alice F., Davis, Roland W., Barry J.