Gene therapy in ophthalmology: validation on cultured retinal cells and explants from postmortem human eyes.

abstract：:In summary, I will reiterate the five points I would like to leave with you today: First, the biological revolution has extraordinary power to do good. As long as the use of our new genetic knowledge is guided by the traditional ideals of the healing professions--to help improve the human condition without doing harm-...

journal_title：Human gene therapy

authors： Healy B

更新日期：1992-02-01 00:00:00

abstract：:Adoptive cellular therapy provides the promise of a potentially powerful general treatment for cancer. Although this is a complex and challenging field, there have been major advances in basic and translational research resulting in clinical trial activity that is now beginning to confirm this promise. However, these ...

journal_title：Human gene therapy

authors： Hawkins RE,Gilham DE,Debets R,Eshhar Z,Taylor N,Abken H,Schumacher TN,ATTACK Consortium

更新日期：2010-06-01 00:00:00

abstract：:Despite efforts toward improvements in retrovirus-mediated gene transfer, stable high-level expression of a therapeutic gene in human hematopoietic stem cells remains a great challenge. We have evaluated the efficiency of different viral long terminal repeats (LTRs) in long-term expression of a transgene in vivo, usin...

journal_title：Human gene therapy

authors： Kaneko S,Onodera M,Fujiki Y,Nagasawa T,Nakauchi H

更新日期：2001-01-01 00:00:00

abstract：:Umbilical cord blood (CB) from the early gestational human fetus is recognized as a rich source of hematopoietic stem cells. To examine the value of fetal CB for gene therapy of inborn immunohematopoietic disorders, we tested the feasibility of genetic modification of CD34(+) cells from CB at weeks 24 to 34 of pregnan...

journal_title：Human gene therapy

authors： Luther-Wyrsch A,Costello E,Thali M,Buetti E,Nissen C,Surbek D,Holzgreve W,Gratwohl A,Tichelli A,Wodnar-Filipowicz A

更新日期：2001-03-01 00:00:00

abstract：:Evaluation of the potential role of dendritic cells (DCs) as adjuvants for tumor vaccination has focused primarily on techniques that load DCs with peptide tumor antigens. Our aim has been to optimize the induction of antitumor immunity by enhancing the ability of DCs to present tumor-associated antigens endogenously ...

journal_title：Human gene therapy

authors： Wan Y,Bramson J,Carter R,Graham F,Gauldie J

更新日期：1997-07-20 00:00:00

abstract：:Due to both the avascularity of the cornea and the relatively immune-privileged status of the eye, corneal transplantation is one of the most successful clinical transplant procedures. However, in high-risk patients, which account for >20% of the 180,000 transplants carried out worldwide each year, the rejection rate ...

journal_title：Human gene therapy

authors： Fouladi N,Parker M,Kennedy V,Binley K,McCloskey L,Loader J,Kelleher M,Mitrophanous KA,Stout JT,Ellis S

更新日期：2018-06-01 00:00:00

abstract：:Advances in cell and gene therapy are opening up new avenues for regenerative medicine. Because of their acquired pluripotency, human induced pluripotent stem cells (hiPSCs) are a promising source of autologous cells for regenerative medicine. They show unlimited self-renewal while retaining the ability, in principle,...

journal_title：Human gene therapy

authors： Garate Z,Davis BR,Quintana-Bustamante O,Segovia JC

更新日期：2013-06-01 00:00:00

abstract：:Artemis is a hairpin-opening endonuclease involved in nonhomologous end-joining and V(D)J recombination. Deficiency of Artemis results in radiation-sensitive severe combined immunodeficiency (SCID) characterized by complete absence of T and B cells due to an arrest at the receptor recombination stage. We have generate...

journal_title：Human gene therapy

authors： Multhaup M,Karlen AD,Swanson DL,Wilber A,Somia NV,Cowan MJ,McIvor RS

更新日期：2010-07-01 00:00:00

abstract：:Combining chemotherapy and immunotherapy is problematic because chemotherapy can ablate the immune responses initiated by modulators of the immune system. We hypothesized that protection of immunocompetent cells from the toxic effects of chemotherapy, using drug resistance gene therapy strategies, would allow the comb...

journal_title：Human gene therapy

authors： McMillin DW,Hewes B,Gangadharan B,Archer DR,Mittler RS,Spencer HT

更新日期：2006-08-01 00:00:00

abstract：:Cell therapies are treatments in which stem or progenitor cells are stimulated to differentiate into specialized cells able to home to and repair damaged tissues. After their discovery, endothelial progenitor cells (EPCs) stimulated worldwide interest as possible vehicles to perform autologous cell therapy of tumors. ...

journal_title：Human gene therapy

authors： Laurenzana A,Margheri F,Chillà A,Biagioni A,Margheri G,Calorini L,Fibbi G,Del Rosso M

更新日期：2016-10-01 00:00:00

abstract：:Less than 20% of the protein coding genome is thought to be targetable using small molecules. mRNA therapies are not limited in the same way since in theory, they can silence or edit any gene by encoding CRISPR nucleases, or alternatively, produce any missing protein. Yet not all mRNA therapies are equally likely to s...

journal_title：Human gene therapy

authors： Da Silva Sanchez A,Paunovska K,Cristian A,Dahlman JE

更新日期：2020-09-01 00:00:00

abstract：:We reported total correction of blood coagulation plasma factor VIII (FVIII) activity, using adeno-associated virus serotype 8 (AAV8) vectors for liver-specific gene transfer in hemophilia A mice. We now show, irrespective of immunosuppression or route of administration, total long-term correction of hemophilia A mice...

journal_title：Human gene therapy

authors： Sarkar R,Mucci M,Addya S,Tetreault R,Bellinger DA,Nichols TC,Kazazian HH Jr

更新日期：2006-04-01 00:00:00

abstract：:Liver ischemia-reperfusion (I/R) injury is a multifactorial process that affects graft function after liver transplantation. Inflammatory cytokines, such as tumor necrosis factor (TNF)-α, interleukin (IL)-6, IL-1β, and IL-18, have been shown to play key roles in the pathophysiology of liver I/R injury. Studies have in...

journal_title：Human gene therapy

authors： Zhu P,Duan L,Chen J,Xiong A,Xu Q,Zhang H,Zheng F,Tan Z,Gong F,Fang M

更新日期：2011-07-01 00:00:00

abstract：:Lentiviral vectors hold great promise for the genetic correction of various inherited diseases. However, lentiviral vector biology is still not completely understood and warrants the precise decoding of molecular mechanisms underlying integration and post-translational modification. This study investigated a series of...

journal_title：Human gene therapy

authors： Scholz SJ,Fronza R,Bartholomä CC,Cesana D,Montini E,von Kalle C,Gil-Farina I,Schmidt M

更新日期：2017-10-01 00:00:00

abstract：:Hematopoietic stem cells (HSCs) are a potential target for the retrovirus-mediated transfer of chemotherapeutic drug resistance genes. For integration of the proviral DNA in the HSC genome cell division is required. In the bone marrow (BM) hematopoiesis occurs in the vicinity of stroma cells. Soluble stroma components...

journal_title：Human gene therapy

authors： Schiedlmeier B,Buss EC,Veldwijk MR,Zeller WJ,Fruehauf S

更新日期：1999-06-10 00:00:00

abstract：:Leukocyte adhesion deficiency type I (LAD-I) is a primary immunodeficiency caused by mutations in the ITGB2 gene and is characterized by recurrent and life-threatening bacterial infections. These mutations lead to defective or absent expression of β2 integrins on the leukocyte surface, compromising adhesion and extrav...

journal_title：Human gene therapy

authors： Leon-Rico D,Aldea M,Sanchez-Baltasar R,Mesa-Nuñez C,Record J,Burns SO,Santilli G,Thrasher AJ,Bueren JA,Almarza E

更新日期：2016-09-01 00:00:00

abstract：:Eleven patients with moderate to severe erectile dysfunction (ED) were given a single-dose corpus cavernosum injection of hMaxi-K, a "naked" DNA plasmid carrying the human cDNA encoding hSlo (for human slow-poke), the gene for the alpha, or pore-forming, subunit of the human smooth muscle Maxi-K channel. Three patient...

journal_title：Human gene therapy

authors： Melman A,Bar-Chama N,McCullough A,Davies K,Christ G

更新日期：2006-12-01 00:00:00

abstract：:SV40 is an attractive potential vector with high-efficiency gene transfer into a wide variety of human tissues, including the bone marrow, a critical target organ for the cure of many diseases. In the present study, the three SV40 capsid proteins, VP1, VP2, and VP3, were produced in Spodoptera frugiperda (Sf9) insect ...

journal_title：Human gene therapy

authors： Sandalon Z,Dalyot-Herman N,Oppenheim AB,Oppenheim A

更新日期：1997-05-01 00:00:00

abstract：:Recombinant adeno-associated virus 2 (rAAV2) has been extensively used as a gene delivery vector for the nervous system. It targets primarily neurons in the nervous system and results in sustained long-term expression of transgenes. New rAAV serotypes have been characterized and demonstrated to have improved transduct...

journal_title：Human gene therapy

authors： Burger C,Nash K,Mandel RJ

更新日期：2005-07-01 00:00:00

abstract：:Sphingosine kinase 1 (SPK1) has been identified as a central mediator of ischemia preconditioning and plays a protective role in ischemia/reperfusion (I/R)-induced cardiomyocyte death. In the present study, we investigated the protective effect of adenovirus-mediated SPK1 gene (Ad-SPK1) transfer on I/R-induced cardiac...

journal_title：Human gene therapy

authors： Duan HF,Wang H,Yi J,Liu HJ,Zhang QW,Li LB,Zhang T,Lu Y,Wu CT,Wang LS

更新日期：2007-11-01 00:00:00

abstract：:Immunotherapy with whole cell cancer vaccines has been tested in various tumor types. This study investigated the safety profile and antitumor activity of an allogeneic prostate carcinoma cell line, LNCaP, expressing recombinant human interleukin-2 and human interferon-gamma. Thirty HLA-A*0201-matched patients with pr...

journal_title：Human gene therapy

authors： Brill TH,Kübler HR,Pohla H,Buchner A,Fend F,Schuster T,van Randenborgh H,Paul R,Kummer T,Plank C,Eisele B,Breul J,Hartung R,Schendel DJ,Gansbacher B

更新日期：2009-12-01 00:00:00

abstract：:Therapeutic levels of expression of the beta-globin gene have been difficult to achieve with conventional retroviral vectors without the inclusion of DNase I-hypersensitive site (HS2, HS3, and HS4) enhancer elements. We generated recombinant adeno-associated viral (AAV) vectors carrying an antisickling human beta-glob...

journal_title：Human gene therapy

authors： Maina N,Zhong L,Li X,Zhao W,Han Z,Bischof D,Aslanidi G,Zolotukhin S,Weigel-Van Aken KA,Rivers AE,Slayton WB,Yoder MC,Srivastava A

更新日期：2008-04-01 00:00:00

abstract：:Glycogen storage disease type II (GSDII) is a lysosomal storage disease caused by a deficiency in acid alpha-glucosidase (GAA), and leads to cardiorespiratory failure by the age of 2 years. In this study, we investigate the impact of anti-GAA antibody formation on cross-correction of the heart, diaphragm, and hind-lim...

journal_title：Human gene therapy

authors： Cresawn KO,Fraites TJ,Wasserfall C,Atkinson M,Lewis M,Porvasnik S,Liu C,Mah C,Byrne BJ

更新日期：2005-01-01 00:00:00

abstract：:Adeno-associated viral (AAV) vectors are becoming increasingly popular in basic research as well as in clinical gene therapy. Due to its exceptional resistance against physical and chemical stress, however, the increasing use of AAV in laboratories and clinics around the globe raises safety concerns. Proper decontamin...

journal_title：Human gene therapy

authors： Korte J,Mienert J,Hennigs JK,Körbelin J

更新日期：2020-11-06 00:00:00

abstract：:Oncolytic measles virus (MV) encoding the human thyroidal sodium iodide symporter (MV-NIS) has proved to be safe after intraperitoneal or intravenous administration in patients with ovarian cancer or multiple myeloma, respectively, but it has not yet been administered through intratumoral injection in humans. Squamous...

journal_title：Human gene therapy

authors： Li H,Peng KW,Russell SJ

更新日期：2012-03-01 00:00:00

abstract：:Cell encapsulation offers a safe and manufacturable method for the systemic delivery of therapeutic proteins from genetically engineered cells. However, control of dose delivery remains a major issue with regard to clinical application. We generated populations of immortalized murine NIH 3T3 fibroblasts that secrete m...

journal_title：Human gene therapy

authors： Serguera C,Bohl D,Rolland E,Prevost P,Heard JM

更新日期：1999-02-10 00:00:00

abstract：:T lymphocytes, regardless of their specificity, are considered key targets for genetic modification in the treatment of inherited or acquired human diseases. In this study, we generated Lewis T cell lines specific for Dark Agouti rat alloantigens and tested the potential of allospecific T lymphocytes as carriers of ge...

journal_title：Human gene therapy

authors： Hammer MH,Flügel A,Seifert M,Lehmann M,Brandt C,Volk HD,Ritter T

更新日期：2000-06-10 00:00:00

abstract：:This Phase I study, "Ribozyme Gene Therapy of HIV-1 Infection" (UCSD HSC #971072, FDA BB-IND 6405), is a prospective, open-label trial of infusion of autologous gene-altered cells into asymptomatic HIV-1 seropositive individuals. The objectives of this trial are to test the safety, feasibility, and potential efficacy ...

journal_title：Human gene therapy

authors： Wong-Staal F,Poeschla EM,Looney DJ

更新日期：1998-11-01 00:00:00

abstract：:Establishing pharmacological parameters, such as efficacy, routes of administration, and toxicity, for recombinant adeno-associated virus (rAAV) vectors is a prerequisite for gaining acceptance for clinical applications. In fact, even a therapeutic window, that is, the dose range between therapeutic efficacy and toxic...

journal_title：Human gene therapy

authors： Virag T,Cecchini S,Kotin RM

更新日期：2009-08-01 00:00:00

abstract：:The transfer of a drug resistance gene into hematopoietic cells is an approach being investigated to overcome the problem of myelosuppression produced by anticancer drugs. Chemotherapeutic agents are often given in combination in order to increase their effectiveness. Consequently, there is an advantage in designing v...

journal_title：Human gene therapy

authors： Beauséjour CM,Le NL,Létourneau S,Cournoyer D,Momparler RL

更新日期：1998-11-20 00:00:00