Authors: Jacobs, Louisa¹, Lambrix, Ellen¹, Smith, Claire¹

Superior Title: Licensing Journal. Apr2024, Vol. 44 Issue 4, p1-5. 5p.

Subject Terms: *DRUG patents, *WARRANTY, *MEDICAL personnel, GENE therapy, CELLULAR therapy, CRITICALLY ill, STEM cell donors, SPINAL muscular atrophy

Authors: Shokati Eshkiki, Zahra¹, Mansouri, Fatemeh², Karamzadeh, Amir Reza², Namazi, Abolfazl^3,4, Heydari, Hafez⁵, Akhtari, Javad⁶, Tabaeian, Seidamir Pasha^3,4, Akbari, Abolfazl^3,7

Superior Title: Journal of Clinical Pharmacy & Therapeutics. 4/5/2024, Vol. 2024, p1-19. 19p.

Subject Terms: *GASTROINTESTINAL tumors, *GENE therapy, *CARBOHYDRATES, *RADIOTHERAPY, *CANCER relapse, *ANTINEOPLASTIC agents, *STRATEGIC planning, *POLYSACCHARIDES, *CANCER chemotherapy, *BIOMEDICAL materials, *BIODEGRADABLE materials, *NANOPARTICLES

Authors: Brotto, Davide^1,2 (AUTHOR) davide.brotto@unipd.it, Greggio, Marco^1,2 (AUTHOR) cosimo.defilippis@unipd.it, De Filippis, Cosimo¹ (AUTHOR) patrizia.trevisi@unipd.it, Trevisi, Patrizia^1,2 (AUTHOR)

Superior Title: Audiology Research. Apr2024, Vol. 14 Issue 2, p239-253. 15p.

Subject Terms: *GENE therapy, *DEAFNESS, *SENSORINEURAL hearing loss, *RECESSIVE genes, *GENETIC mutation, *HEARING disorders, *BONE conduction

Authors: Moyu Dai^1,2,3, Ning Yang^1,2,3, Kai Xu^1,2,4, Jingwen Zhang^1,5, Xueke Li^1,2,3, Ying Zhang^1,2,3,4, Wei Li^1,2,3,4 liwei@ioz.ac.cn

Superior Title: Cell Proliferation. Mar2024, Vol. 57 Issue 3, p1-11. 11p.

Subject Terms: *GENE therapy, *LABORATORY mice, *HUMAN genes, *GENETIC vectors, *ADENO-associated virus, *LIVER cells, *ADENOVIRUSES, *RHABDOVIRUSES

Authors: Hadi, Meead¹ (AUTHOR), Qutaiba B. Allela, Omer² (AUTHOR), Jabari, Mansoureh³ (AUTHOR), Jasoor, Asna Mahyazadeh⁴ (AUTHOR), Naderloo, Omid⁵ (AUTHOR), Yasamineh, Saman⁶ (AUTHOR) Yassaman124@gmail.com, Gholizadeh, Omid⁶ (AUTHOR) ogholizade1374@gmail.com, Kalantari, Leila⁷ (AUTHOR) Leilakln284@gmail.com

Superior Title: Virology Journal. 1/12/2024, Vol. 21 Issue 1, p1-22. 22p.

Subject Terms: *ADENO-associated virus, *GENE therapy, *HEPATOCELLULAR carcinoma, *THERAPEUTICS, *LIFE cycles (Biology), *GENETIC transformation, *BOVINE viral diarrhea virus, *COMPREHENSION in children

Authors: Polek, Hubert¹ hpo@assignity.com, Janik, Justyna², Paterak, Ewelina², Dabbous, Monique³, Pochopień, Michał³, Toumi, Mondher³

Superior Title: Journal of Market Access & Health Policy. Dec2023, Vol. 11 Issue 1, p1-7. 7p.

Subject Terms: *AMORTIZATION, *BUDGET, *PRICES, GENE therapy, TIME perspective

Authors: Liu, Jian¹ (AUTHOR), Zheng, Hui¹ (AUTHOR), Wei, Li² (AUTHOR)

Superior Title: Journal of Immunology Research. 12/9/2023, p1-28. 28p.

Subject Terms: *DISEASE risk factors, *GENE therapy, *PROGNOSTIC models, *IMMUNOTHERAPY, *SQUAMOUS cell carcinoma

Authors: Hahn, Roni¹ (AUTHOR) hahnroni@mail.tau.ac.il, Avraham, Karen B.¹ (AUTHOR) karena@tauex.tau.ac.il

Superior Title: Audiology Research. Dec2023, Vol. 13 Issue 6, p952-966. 15p.

Subject Terms: *GENE therapy, *HEARING disorders, *HEARING aids, *COCHLEAR implants, *DEAFNESS

Authors: Smith, Gerry, Contiliano, Tom

Superior Title: Bloomberg Businessweek. 2/26/2024, Issue 4813, p10-12. 3p. 1 Graph, 1 Cartoon or Caricature.

Subject Terms: *HEALTH facilities, GENE therapy, SPOKESPERSONS, SEVERE combined immunodeficiency, SPINAL muscular atrophy

Company/Entity: BIOMARIN Pharmaceutical Inc. BMRN

Authors: Shuman, Tristan¹ (AUTHOR)

Superior Title: Epilepsy Currents. Feb2024, Vol. 24 Issue 1, p56-58. 3p.

Subject Terms: *GENE therapy, *EPILEPSY, *NEURONS, *POTASSIUM channels, *NEUROBEHAVIORAL disorders

Authors: Herring, William L.^1,2 (AUTHOR) wherring@rti.org, Gallagher, Meghan E.³ (AUTHOR), Shah, Nirmish⁴ (AUTHOR), Morse, KC⁵ (AUTHOR), Mladsi, Deirdre¹ (AUTHOR), Dong, Olivia M.¹ (AUTHOR), Chawla, Anjulika⁶ (AUTHOR), Leiding, Jennifer W.⁷ (AUTHOR), Zhang, Lixin⁸ (AUTHOR), Paramore, Clark⁹ (AUTHOR), Andemariam, Biree¹⁰ (AUTHOR)

Superior Title: PharmacoEconomics. Jun2024, Vol. 42 Issue 6, p693-714. 22p.

Subject Terms: *COST effectiveness, SICKLE cell anemia, GENE therapy, HEMATOPOIETIC stem cell transplantation, BURDEN of care

Geographic Terms: UNITED States

Authors: Barton, Cheryl¹ info@pharmavision.co.uk

Superior Title: Pharmaceutical Technology Europe. May2024, Vol. 36 Issue 5, p15-19. 4p.

Subject Terms: *DIFFUSION of innovations, DRUG therapy for Parkinson's disease, GENE therapy, PHARMACEUTICAL technology, GLIOMAS, ALZHEIMER'S disease, BLOOD-brain barrier, CENTRAL nervous system, DRUG delivery systems, NEURODEGENERATION, MONOCLONAL antibodies, DRUG approval, PHARMACY information services, DRUG development

Company/Entity: UNITED States. Food & Drug Administration

Authors: Campochiaro, Peter A¹ (AUTHOR) pcampo@jhmi.edu, Avery, Robert² (AUTHOR), Brown, David M³ (AUTHOR), Heier, Jeffrey S⁴ (AUTHOR), Ho, Allen C⁵ (AUTHOR), Huddleston, Stephen M⁶ (AUTHOR), Jaffe, Glenn J⁷ (AUTHOR), Khanani, Arshad M⁸ (AUTHOR), Pakola, Stephen⁹ (AUTHOR), Pieramici, Dante J² (AUTHOR), Wykoff, Charles C³ (AUTHOR), Van Everen, Sherri⁹ (AUTHOR)

Superior Title: Lancet. Apr2024, Vol. 403 Issue 10436, p1563-1573. 11p.

Subject Terms: MACULAR degeneration, GENE therapy, ENDOTHELIAL growth factors, AQUEOUS humor, VISUAL acuity

Authors: Broadfoot, Marla

Superior Title: Scientific American. Nov2021, Vol. 325 Issue 5, pS15-S19. 5p. 2 Color Photographs.

Subject Terms: PATIENTS, GENE therapy, ERYTHROCYTES, BLOOD vessels, SPINAL muscular atrophy

Authors: Nyati, Shyam^1,2 (AUTHOR) snyati1@hfhs.org, Stricker, Hans³ (AUTHOR), Barton, Kenneth N.¹ (AUTHOR), Li, Pin⁴ (AUTHOR), Elshaikh, Mohamed¹ (AUTHOR), Ali, Haythem⁵ (AUTHOR), Brown, Stephen L.^1,6 (AUTHOR), Hwang, Clara⁵ (AUTHOR), Peabody, James³ (AUTHOR), Freytag, Svend O.¹ (AUTHOR), Movsas, Benjamin^1,6 (AUTHOR), Siddiqui, Farzan¹ (AUTHOR)

Superior Title: PLoS ONE. 9/15/2023, Vol. 18 Issue 9, p1-22. 22p.

Subject Terms: *ADENOVIRUSES, *GENE therapy, *MONONUCLEAR leukocytes, *INTERLEUKIN-12, *CLINICAL trials, *PROSTATE

Authors: Son, Jin Sung¹ (AUTHOR), Chow, Ryan² (AUTHOR), Kim, Helena¹ (AUTHOR), Lieu, Toney¹ (AUTHOR), Xiao, Maria¹ (AUTHOR), Kim, Sunny¹ (AUTHOR), Matuszewska, Kathy³ (AUTHOR), Pereira, Madison³ (AUTHOR), Nguyen, David Le² (AUTHOR), Petrik, Jim^1,3,4 (AUTHOR) jpetrik@uoguelph.ca

Superior Title: Reproductive Biology & Endocrinology. 8/23/2023, Vol. 21 Issue 1, p1-11. 11p.

Subject Terms: *OVARIAN cancer, *GENE therapy, *MITOGEN-activated protein kinases, *CATIONIC lipids, *CANCER treatment, *EPHRIN receptors, *TUMOR suppressor genes

Authors: Mikkelsen, Nanna S.¹ (AUTHOR), Bak, Rasmus O.¹ (AUTHOR) bak@biomed.au.dk

Superior Title: Journal of Biomedical Science. 7/1/2023, Vol. 30 Issue 1, p1-21. 21p.

Subject Terms: *GENOME editing, *GENE therapy, *GENOMICS, *CELLULAR therapy, *CRISPRS

Authors: Wilson, Clare (AUTHOR)

Superior Title: New Scientist. 2/10/2024, Vol. 261 Issue 3477, p18-18. 2/3p.

Subject Terms: *GENE therapy, *CRISPRS, *SICKLE cell anemia

Authors: Schambach, Axel^1,2,3,4 (AUTHOR), Buchholz, Christian J^5,6 (AUTHOR), Torres-Ruiz, Raul^7,8,9 (AUTHOR), Cichutek, Klaus⁵ (AUTHOR), Morgan, Michael¹ (AUTHOR), Trapani, Ivana^10,11 (AUTHOR), Büning, Hildegard^1,3,4 (AUTHOR) buening.hildegard@mh-hannover.de

Superior Title: Lancet. Feb2024, Vol. 403 Issue 10426, p568-582. 15p.

Subject Terms: GENE therapy, GENOME editing, CLINICAL trials

Authors: Zemplenyi, Antal^1,2 (AUTHOR) antal.zemplenyi@cuanschutz.edu, Leonard, Jim³ (AUTHOR), DiStefano, Michael J.¹ (AUTHOR), Anderson, Kelly E.¹ (AUTHOR), Wright, Garth C.¹ (AUTHOR), Mendola, Nicholas D.¹ (AUTHOR), Nair, Kavita¹ (AUTHOR), McQueen, R. Brett¹ (AUTHOR)

Superior Title: PharmacoEconomics. Mar2024, Vol. 42 Issue 3, p319-328. 10p.

Subject Terms: *MEDICAID, *MEDICAID costs, GENE therapy, BLOOD coagulation factor IX, HEALTH policy