Volume 25; Issue 7

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Human Gene Therapy
Volume 25; Issue 7

Human Gene Therapy

Volume 25; Issue 7

1

Similar Therapeutic Efficacy Between a Single Administration of Gene Therapy and Multiple Administrations of Recombinant Enzyme in a Mouse Model of Lysosomal Storage Disease

Ferla, Rita, Claudiani, Pamela, Cotugno, Gabriella, Saccone, Paola, De Leonibus, Elvira, Auricchio, Alberto

Journal:

Human Gene Therapy

Année:

2014

Langue:

english

Fichier:

PDF, 511 KB

english, 2014

2

Gene Therapy: A Promising Approach to Treating Spinal Muscular Atrophy

Mulcahy, Pádraig J., Iremonger, Kayleigh, Karyka, Evangelia, Herranz-Martín, Saúl, Shum, Ka-To, Tam, Janice Kal Van, Azzouz, Mimoun

Journal:

Human Gene Therapy

Année:

2014

Langue:

english

Fichier:

PDF, 1.08 MB

english, 2014

3

Small Interfering RNAs Targeted to Interleukin-4 and Respiratory Syncytial Virus Reduce Airway Inflammation in a Mouse Model of Virus-Induced Asthma Exacerbation

Khaitov, Musa R., Shilovskiy, Igor P., Nikonova, Aleksandra A., Shershakova, Nadezda N., Kamyshnikov, Oleg Y., Babakhin, Alexander A., Zverev, Vitaly V., Johnston, Sebastian L., Khaitov, Rakhim M.

Journal:

Human Gene Therapy

Année:

2014

Langue:

english

Fichier:

PDF, 507 KB

english, 2014

4

Gene-Modified Cells for Stem Cell Transplantation and Cancer Therapy

Brenner, Malcolm K.

Journal:

Human Gene Therapy

Année:

2014

Langue:

english

Fichier:

PDF, 154 KB

english, 2014

5

Phenotypic Reversion of Fair Hair upon Gene Therapy of the Phenylketonuria Mice

Thöny, Beat, Ding, Zhaobing, Rebuffat, Alexandre, Viecelli, Hiu Man

Journal:

Human Gene Therapy

Année:

2014

Langue:

english

Fichier:

PDF, 374 KB

english, 2014

6

Translational Fidelity of Intrathecal Delivery of Self-Complementary AAV9–Survival Motor Neuron 1 for Spinal Muscular Atrophy

Passini, Marco A., Bu, Jie, Richards, Amy M., Treleaven, Christopher M., Sullivan, Jennifer A., O'Riordan, Catherine R., Scaria, Abraham, Kells, Adrian P., Samaranch, Lluis, San Sebastian, Waldy, Fede

Journal:

Human Gene Therapy

Année:

2014

Langue:

english

Fichier:

PDF, 1.51 MB

english, 2014

7

Development of Therapeutic Splice-Switching Oligonucleotides

Disterer, Petra, Kryczka, Adrianna, Liu, Yuqi, Badi, Yusef E., Wong, Jessie J., Owen, James S., Khoo, Bernard

Journal:

Human Gene Therapy

Année:

2014

Langue:

english

Fichier:

PDF, 478 KB

english, 2014

8

Transgenic Expression of Human Glial Cell Line-Derived Neurotrophic Factor from Integration-Deficient Lentiviral Vectors is Neuroprotective in a Rodent Model of Parkinson's Disease

Lu-Nguyen, Ngoc B., Broadstock, Martin, Schliesser, Maximilian G., Bartholomae, Cynthia C., von Kalle, Christof, Schmidt, Manfred, Yáñez-Muñoz, Rafael J.

Journal:

Human Gene Therapy

Année:

2014

Langue:

english

Fichier:

PDF, 1.17 MB

english, 2014

9

Congratulations to the Winners of the 12 Pioneer Awards for Seminal Work in Gene and Cell Therapy

Journal:

Human Gene Therapy

Année:

2014

Langue:

english

Fichier:

PDF, 808 KB

english, 2014

10

Characterization of Vector-Based Delivery of Neurogenin-3 in Murine Diabetes

Phillips, Neil, Kay, Mark A.

Journal:

Human Gene Therapy

Année:

2014

Langue:

english

Fichier:

PDF, 682 KB

english, 2014

11

Gene Therapy Briefs

Philippidis, Alex

Journal:

Human Gene Therapy

Année:

2014

Langue:

english

Fichier:

PDF, 69 KB

english, 2014

12

Dissecting the Mechanism of Histone Deacetylase Inhibitors to Enhance the Activity of Zinc Finger Nucleases Delivered by Integrase-Defective Lentiviral Vectors

Joglekar, Alok V., Stein, Libby, Ho, Michelle, Hoban, Megan D., Hollis, Roger P., Kohn, Donald B.

Journal:

Human Gene Therapy

Année:

2014

Langue:

english

Fichier:

PDF, 488 KB

english, 2014