Evidence map›Paper›PMID 10673714›Full record

ReviewGene therapy2000

Gene therapy in the CNS.

L C Costantini, J C Bakowska, X O Breakefield, O Isacson

Open access · bronzeAbstract readReview
PubMed Publisher
In one paragraph

Review in Gene therapy, 2000. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 36 papers.

0numbers the graph read from it
0cells of the map it votes in
36citing papers in PubMed
10.6field-weighted citation impact, top 1% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

36 citing papers in PubMed, 141 citations in OpenAlex.

  1. Article
  2. Review
  3. Review
  4. Dual-Modified Liposome for Targeted and Enhanced Gene Delivery into Mice Brain.The Journal of pharmacology and experimental therapeutics · 2020
    Article
  5. Article
  6. MRI-Guided Focused Ultrasound for Targeted Delivery of rAAV to the Brain.Methods in molecular biology (Clifton, N.J.) · 2019
    Article
  7. Article
  8. Review
  9. Article
  10. Article
  11. Article
  12. Article
  13. Antioxidant-based therapies for angiotensin II-associated cardiovascular diseases.American journal of physiology. Regulatory, integrative and comparative physiology · 2013
    Review
  14. Article
  15. Article
  16. Article
  17. Article
  18. Lentiviral vectors in gene therapy: their current status and future potential.Archivum immunologiae et therapiae experimentalis · 2010
    Review
  19. Article
  20. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

4 authors at 2 institutions in 1 country.

L C CostantiniNeuroregeneration Laboratory, Harvard Medical School, McLean Hospital, Belmont, MA, USA.
J C Bakowska
X O Breakefield
O Isacson
Harvard University · USMcLean Hospital · US

Funding

Vector Query and Therapy For TSC and NF2 LesionsP01NS024279 · NINDS · MASSACHUSETTS GENERAL HOSPITAL · PI KWIATKOWSKI, DAVID J. · 1987 to 2012
$14.7M
HSV/AAV HYBRID VECTORS FOR GENE REPLACEMENTR21MH060587 · NIMH · MASSACHUSETTS GENERAL HOSPITAL · PI BREAKEFIELD, XANDRA OWENS · 1999 to 2000
$170k
CLINICAL NEUROSCIENCE TRAINING PROGRAM FOR HARVARDT32MH019905 · NIMH · MC LEAN HOSPITAL (BELMONT, MA) · PI BENES, FRANCINE M. · 1994 to 1998
–
NIMH NIH HHS R21MH60587NIMH NIH HHS T32 MH 19905NINDS NIH HHS NS24279
6 · The paper itself

Abstract

Gene therapy for neurological disorder is currently an experimental concept. The goals for clinical utilization are the relief of symptoms, slowing of disease progression, and correction of genetic abnormalities. Experimental studies are realizing these goals in the development of gene therapies in animal models. Discoveries of the molecular basis of neurological disease and advances in gene transfer systems have allowed focal and global delivery of therapeutic genes for a wide variety of CNS disorders. Limitations are still apparent, such as stability and regulation of transgene expression, and safety of both vector and expressed transgene. In addition, the brain adds several challenges not seen in peripheral gene therapy paradigms, such as post-mitotic cells, heterogeneity of cell types and circuits, and limited access. Moreover, it is likely that several modes of gene delivery will be necessary for successful gene therapies of the CNS. Collaborative efforts between clinicians and basic researchers will likely yield effective gene therapy in the CNS.

Indexed as

Central Nervous System DiseasesGenetic TherapyGenetic VectorsGene Transfer TechniquesHerpesvirus 4, HumanHumansLentivirusRetroviridaeSimplexvirus

Identifiers

PMID10673714
OpenAlexW1997034660

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.