Volume 23; Issue 4

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Human Gene Therapy
Volume 23; Issue 4

Human Gene Therapy

Volume 23; Issue 4

1

Long-Term Expression of Human Coagulation Factor VIII in a Tolerant Mouse Model Using the φC31 Integrase System

Chavez, Christopher L., Keravala, Annahita, Chu, Jacqueline N., Farruggio, Alfonso P., Cuéllar, Vanessa E., Voorberg, Jan, Calos, Michele P.

Journal:

Human Gene Therapy

Year:

2012

Language:

english

File:

PDF, 336 KB

english, 2012

2

Adeno-Associated Virus Serotype 9 Transduction in the Central Nervous System of Nonhuman Primates

Samaranch, Lluis, Salegio, Ernesto A., San Sebastian, Waldy, Kells, Adrian P., Foust, Kevin D., Bringas, John R., Lamarre, Clementine, Forsayeth, John, Kaspar, Brian K., Bankiewicz, Krystof S.

Journal:

Human Gene Therapy

Year:

2012

Language:

english

File:

PDF, 1.15 MB

english, 2012

3

Dextran and Protamine-Based Solid Lipid Nanoparticles as Potential Vectors for the Treatment of X-Linked Juvenile Retinoschisis

Delgado, Diego, del Pozo-Rodríguez, Ana, Solinís, Maria Ángeles, Avilés-Triqueros, Marcelino, Weber, Bernhard H.F., Fernández, Eduardo, R. Gascón, Alicia

Journal:

Human Gene Therapy

Year:

2012

Language:

english

File:

PDF, 1.01 MB

english, 2012

4

Long-Term Rescue of Retinal Structure and Function by Rhodopsin RNA Replacement with a Single Adeno-Associated Viral Vector in P23H RHO Transgenic Mice

Mao, Haoyu, Gorbatyuk, Marina S., Rossmiller, Brian, Hauswirth, William W., Lewin, Alfred S.

Journal:

Human Gene Therapy

Year:

2012

Language:

english

File:

PDF, 785 KB

english, 2012

5

Successful Gene Therapy in Utero for Lethal Murine Hypophosphatasia

Sugano, Hanako, Matsumoto, Tae, Miyake, Koichi, Watanabe, Atsushi, Iijima, Osamu, Migita, Makoto, Narisawa, Sonoko, Millán, José Luis, Fukunaga, Yoshitaka, Shimada, Takashi

Journal:

Human Gene Therapy

Year:

2012

Language:

english

File:

PDF, 504 KB

english, 2012

6

Long-Term Efficacy Following Readministration of an Adeno-Associated Virus Vector in Dogs with Glycogen Storage Disease Type Ia

Demaster, Amanda, Luo, Xiaoyan, Curtis, Sarah, Williams, Kyha D., Landau, Dustin J., Drake, Elizabeth J., Kozink, Daniel M., Bird, Andrew, Crane, Bayley, Sun, Francis, Pinto, Carlos R., Brown, Talmage

Journal:

Human Gene Therapy

Year:

2012

Language:

english

File:

PDF, 1.22 MB

english, 2012

7

Gene Therapy for Retinitis Pigmentosa Caused by MFRP Mutations: Human Phenotype and Preliminary Proof of Concept

Dinculescu, Astra, Estreicher, Jackie, Zenteno, Juan C., Aleman, Tomas S., Schwartz, Sharon B., Huang, Wei Chieh, Roman, Alejandro J., Sumaroka, Alexander, Li, Qiuhong, Deng, Wen-Tao, Min, Seok-Hong,

Journal:

Human Gene Therapy

Year:

2012

Language:

english

File:

PDF, 723 KB

english, 2012

8

Effective Radiovirotherapy for Malignant Gliomas by Using Oncolytic Measles Virus Strains Encoding the Sodium Iodide Symporter (MV-NIS)

Opyrchal, Mateusz, Allen, Cory, Iankov, Ianko, Aderca, Ileana, Schroeder, Mark, Sarkaria, Jann, Galanis, Evanthia

Journal:

Human Gene Therapy

Year:

2012

Language:

english

File:

PDF, 635 KB

english, 2012

9

Gene Therapy Briefs

Philippidis, Alex

Journal:

Human Gene Therapy

Year:

2012

Language:

english

File:

PDF, 34 KB

english, 2012

10

Long-Term Evaluation of a Phase 1 Study of AADC Gene Therapy for Parkinson's Disease

Mittermeyer, Gabriele, Christine, Chadwick W., Rosenbluth, Kathryn H., Baker, Suzanne L., Starr, Philip, Larson, Paul, Kaplan, Paul L., Forsayeth, John, Aminoff, Michael J., Bankiewicz, Krystof S.

Journal:

Human Gene Therapy

Year:

2012

Language:

english

File:

PDF, 243 KB

english, 2012

11

Transfection of Naked Nuclear Factor-κB Decoy Oligodeoxynucleotides into Liver by Rapid Portal Vein Infusion in Rats: Its Effect on Ischemia–Reperfusion Injury of Liver

Watanabe, Go, Uchinami, Hiroshi, Yoshioka, Masato, Abe, Yuki, Kikuchi, Isao, Iwasaki, Wataru, Kume, Makoto, Yamamoto, Yuzo

Journal:

Human Gene Therapy

Year:

2012

Language:

english

File:

PDF, 713 KB

english, 2012

12

Gene Therapy for Retinal Dystrophies: Twenty Years in the Making

Journal:

Human Gene Therapy

Year:

2012

Language:

english

File:

PDF, 61 KB

english, 2012

13

Challenges for Gene Therapy of CNS Disorders and Implications for Parkinson's Disease Therapies

Broadstock, Martin, Yáñez-Muñoz, Rafael J.

Journal:

Human Gene Therapy

Year:

2012

Language:

english

File:

PDF, 92 KB

english, 2012

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